Practical Solutions for Today’s Bioprocess Challenges

Practical Solutions for Today’s Bioprocess Challenges

A N N U A L

AUGUST 13-17, 2018SHERATON BOSTON | BOSTON, MA

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PLENARY KEYNOTE SESSION

POSTERS

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Upstream Processing

Downstream Processing

Analytical & Quality

Formulation & Stability

Cell Therapy

Gene Therapy

Manufacturing

TRAINING SEMINARS

SHORT COURSES

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EVENT AT A GLANCE Click on track title to view agenda

Monday - Tuesday (August 13-14) Wednesday - Thursday AM (August 15-16) Thursday PM - Friday (August 16-17)

Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors & Disposables Optimizing Cell Line Development

Continuous Processing in Biopharm Manufacturing* Advances in Purification & Recovery Detection, Characterization and Control of Impurities in Biologics †

Host Cell Proteins Accelerating Analytical Development Process Characterization and Control

DINNER SHORT COURSES Thursday, August 16

Rapid Methods to Assess Quality & Stability of Biologics

DINNER SHORT COURSES Tuesday, August 14

Overcoming Formulation Challenges

SHORT COURSES Monday, August 13

High-Concentration Protein FormulationsDetection, Characterization and Control of Impurities in Biologics †

Cell Therapy CMC and Analytics

Gene Therapy CMC and Analytics Gene Therapy Manufacturing

Cell Therapy Manufacturing

Continuous Processing in Biopharm Manufacturing* Manufacturing 4.0

Introduction to Bioprocessing

Regulatory Requirements across the Product Development Lifecycle

Introduction to Working with CROs, CDMOs, and CMOs

Potency Bioassay: Practical Approaches to Development and Validation

A Line-of-Sight Approach to Continued Process Verification Planning

Introduction to Analytical Method Development and Validation for Therapeutic Proteins

Introduction to Cell Culture

Bioprocess Strategies from Development to BLA

Data Management in Biotherapeutic Analytical and Process Development

Fundamentals of Proteins and Protein Solutions

Introduction to Downstream Processing

Designing Flexible Facilities for Bioprocess Development and Manufacturing

Introduction to Biologics Formulation Development

Design of Experiments for Cell and Gene Therapies

Give yourself a Bioprocess Holiday this summer! Stimulate and enrich yourself at the 10th Annual The Bioprocessing Summit — the leading bioprocess event!

Introduction to Biologics Formulation Development ǂ

Design of Experiments for Cell and Gene Therapies ǂ

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Upstream Processing




Cell Therapy

Gene Therapy

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* Streams 2 & 7; † Streams 2 & 4, ǂTraining Seminar

http://www.bioprocessingsummit.com/cell-culture/

http://www.bioprocessingsummit.com/bioproduction/

http://www.bioprocessingsummit.com/cell-line-development/

http://www.bioprocessingsummit.com/continuous-processing/

http://www.bioprocessingsummit.com/purification-technologies/

http://www.bioprocessingsummit.com/viral-clearance/

http://www.bioprocessingsummit.com/host-cell-proteins/

http://www.bioprocessingsummit.com/preclinical-analytical-development/

http://www.bioprocessingsummit.com/process-characterization/

http://www.bioprocessingsummit.com/stability-biologics/

http://www.bioprocessingsummit.com/formulation-challenges/

http://www.bioprocessingsummit.com/protein-formulations/

http://www.bioprocessingsummit.com/viral-clearance/

http://www.bioprocessingsummit.com/cell-therapy-cmc/

http://www.bioprocessingsummit.com/gene-therapy-cmc/

http://www.bioprocessingsummit.com/gene-therapy-bioproduction/

http://www.bioprocessingsummit.com/cell-therapy-bioproduction/

http://www.bioprocessingsummit.com/continuous-processing/

http://www.bioprocessingsummit.com/manufacturing-efficiencies/

http://www.bioprocessingsummit.com/bpd/training-seminars/





























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YEARSABOUT THE SUMMIT The Bioprocessing Summit convenes more than 1,000 international bioprocess professionals to share practical solutions for today's bioprocess challenges. Now in its tenth year, the event has grown to include 17 distinct conferences with weeklong programming on upstream and downstream processing, analytical development and quality, formulation and stability, cell and gene therapy production, and manufacturing. Along with the impressive array of conferences, the Summit also includes short courses and training seminars that provide in-depth coverage of critical bioprocess topics.

WHO TO EXPECT IN 2018

COMPANY TYPEn Biotech + Pharma 86%n Academic/Government 8%n Services + Societies 3%n Healthcare 2%n Other (Financial, Press) 1%

DELEGATE TITLEn Scientist/Technologist 41%n Executive + Director 29%n Sales & Marketing 16%n Manager 9%n Professor 5%

CompanyType

GEOGRAPHIC LOCATIONn United States 83%n Europe 11%n Asia 4%n Rest of World 2%

USA BREAKDOWNn East Coast 72%n West Coast 20%n Midwest 8%

GeographicLocation

USA

Delegate Title

In 2017, attendees included industry leaders, innovators and decision makers from many different backgrounds

9 8 7 6 5 4 32009 – 2018



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POSTERS

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Upstream Processing




Cell Therapy

Gene Therapy

Manufacturing

TRAINING SEMINARS

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The Next 10 Years: Looking Ahead to Next-Gen Bioprocessing Wednesday, August 15 (4:45 - 6:25 pm)

4:45 pm Chairperson’s RemarksJohn Sterling, Editor in Chief, Genetic Engineering & Biotechnology News (GEN)

4:50 Digital Transformation to Advance Next-Generation Biomanufacturing through Data Integration and AnalyticsJerry Murry, PhD, SVP, Process Development, AmgenHundreds of millions of data points are currently generated through the

development and execution of biopharmaceutical processes. It is expected that the volume and complexity of biomanufacturing data is set to grow exponentially as developers and manufacturers integrate novel sensors, smart materials, process analytical technologies and process automation into laboratories and manufacturing plants. This presentation will describe the value associated with a comprehensive digital strategy incorporating a structured data integration and analytics platform inclusive of AI, predictive modeling and visualization, and how digital transformation can advance next-generation biomanufacturing.

5:20 Driving Change in Biomanufacturing through Innovation in Processes, Technologies and OperationsEliana Clark, PhD, Vice President, International Manufacturing Operations, Biogen

A critical step in meeting the demand of biologic production worldwide involves implementing disruptive manufacturing technologies, processes and capabilities. This talk will use Biogen’s new manufacturing site in Switzerland, due to go online in 2019, as an example to demonstrate the new processes, operational models and technologies being adopted to drive value through innovation and deliver new medicines in areas such as Alzheimer’s.

5:50 End-to-End Solutions Considering New Trends in BiomanufacturingGuillaume Plane, MSc, MBA, Manager, Global Development, Biodevelopment Solutions, MilliporeSigma

The presentation will get into the current state of biomanufacturing, from DNA to market approval, considering the way a key supplier can support drug makers to the fullest, thanks to a deep understanding of the trends that could affect our industry in the midterm, including growth of the pipelines, strengthening of regulations, and acceleration of timelines, for development as well as for the set-up of capabilities. Some thoughts and ideas will be proposed to consider commercial manufacturing with single-use equipment.

6:25 Close of Plenary Keynote Session



COVER

EVENT AT A GLANCE


POSTERS

CONFERENCE STREAMS

Upstream Processing




Cell Therapy

Gene Therapy

Manufacturing

TRAINING SEMINARS

SHORT COURSES


HOTEL & TRAVEL


Register Online!






By Cambridge Healthtech Institute

MONDAY, AUGUST 13 AND TUESDAY, AUGUST 14Day One: 1:00 – 5:00 pm | Day Two: 8:00 am – 5:00 pm

TS1A: Introduction to BioprocessingCHI’s Introduction to Bioprocessing training seminar offers a comprehensive survey of the steps needed to produce today’s complex biopharmaceuticals, from early development through commercial. The seminar begins with a brief introduction to biologic drugs and the aspects of protein science that drive the intricate progression of analytical and process steps that follow. We then step through the stages of bioprocessing, beginning with the development of cell lines and ending at scaling up for commercial production. The seminar also explores emerging process technologies, facility design considerations and the regulatory and quality standards that govern our industry throughout development. The important roles of analytical methods at all stages of development as well as formulation and stability assessments in developing and gaining approval for a biopharmaceutical are also examined. This 1.5-day class is directed to attendees working in any aspect of industry, including scientific, technical, business, marketing or support functions, who would benefit from a detailed overview of this field.Instructors:

Sheila G. Magil, PhD, Senior Consultant, BioProcess Technology Consultants, Inc.

Frank J. Riske, PhD, Senior Consultant, BioProcess Technology Consultants, Inc.

TS2A: Regulatory Requirements across the Product Development LifecycleThe successful development of a pharmaceutical product requires not only good science, but also compliance with FDA regulatory expectations. This course will include a comprehensive review of the Chemistry, Manufacturing and Controls (CMC) section of regulatory filings, with a focus on phase appropriate requirements. The level of detail that must be included in the filing will be discussed as well as systems and controls that must be in place in the manufacturing setting. Topics such as process development, analytical development, Good Manufacturing Practices (GMP) and Good Laboratory Practices (GLP) will be discussed in the context of the stage of drug development. Regulatory strategies for navigating the path to approval will also be discussed. This course is intended to provide participants from all facets of the pharmaceutical and biotech industry with a broad understanding of regulatory requirements across the product development lifecycle.

WEDNESDAY, AUGUST 15 AND THURSDAY, AUGUST 16Day One: 8:00 am – 6:00 pm | Day Two: 8:00 am – 12:15 pm

TS7B: Introduction to Analytical Method Development and Validation for Therapeutic ProteinsThis course is a panoramic review of analytical method development and validation for therapeutic proteins, including antibodies and enzymes. It starts with basic knowledge of work on therapeutic proteins: manufacturing of protein drugs, regulatory affair knowledge and protein chemistry. It then discusses fundamentals and practical aspects of commonly used analytical methods for proteins, including methods for structure elucidation, glycan characterization, biophysical characterization, potency measurement, purity and impurity analysis. The course concludes with the strategy and common practice in method validation and method

transfer, including regulatory compliance at different stages of product development, application of DOE and QbD.Instructor:

Jichao (Jay) Kang, PhD, RAC, Director, Analytical Development, Amicus Therapeutics

TS8B: Introduction to Cell CultureThis 1.5-day Intro to Cell Culture Training Seminar is a lecture-based course intended for the beginner who is thinking about culturing animal cells for the first time or for intermediate cell culturists wanting to know more about how animal cell culture works and how to improve their process. Attendees will learn about most of the critical aspects of cell culture from equipment maintenance and media selection to cell growth and cryopreservation. Participants will have ample time to ask specific questions and get worthwhile answers on such topics as: equipment use and decontamination, cell types, cell verification, maintenance and storage, and strategies for growing animal cells in culture.Instructor:

Timothy Fawcett, PhD, Scientific Director, BioTechnical Institute of Maryland, Inc. and Founder, BioSciConcepts

TS9B: Bioprocess Strategies from Development to BLAToday’s biopharmaceutical development environment is driven by a constant stream of new modalities and product formats, an increased focus on product quality and management imperatives to advance through clinical development rapidly, and at the lowest possible cost. The Bioprocess Strategies training seminar presents a look at the key timelines and strategic considerations during the timeline, and explores strategies at each step that can be employed to meet these important goals. Special consideration will be given to the role of new process and analytical technologies, automation and the use of global manufacturing and supply chain networks.Instructor:

Sheila G. Magil, PhD, Senior Consultant, BioProcess Technology Consultants, Inc.

THURSDAY, AUGUST 16 AND FRIDAY, AUGUST 17Day One: 1:30 – 5:00 pm | Day Two: 8:30 am – 3:30 pm

TS11C: Fundamentals of Proteins and Protein SolutionsA simple energy framework is presented that allows a fundamental, but very practical, understanding of protein structure, folding, stability, interactions and solution behavior. The seminar focuses on the practical understanding and application of the energy framework. Building on a review of basic biochemistry and central energy concepts, the framework is used to build up a deeper understanding of how protein folding and structure arise from the properties of its constituent atoms and amino acids. This same energy framework is used to understand protein interactions with small molecules, surfaces, other proteins, and other macromolecules. The importance of cooperativity to biological processes is discussed.Instructor:

Thomas Laue, PhD, Professor Emeritus, Biochemistry and Molecular Biology; Director, Biomolecular Interaction Technologies Center (BITC), University of New Hampshire



COVER

EVENT AT A GLANCE


POSTERS

CONFERENCE STREAMS

Upstream Processing




Cell Therapy

Gene Therapy

Manufacturing

TRAINING SEMINARS

SHORT COURSES


HOTEL & TRAVEL


Register Online!






By Cambridge Healthtech Institute

TS12C: Introduction to Downstream ProcessingThis activity-packed course focuses on the science, technologies and strategies needed to understand and implement an effective downstream process for biological development and production. The course begins with an in-depth look at DSP design and development - from recovery to purification to formulation - before moving onto pertinent issues surrounding HTPD, single-use systems, continuous processing, PAT, CPPs, viral clearance, platform development and process validation. The course concludes with real-world examples from downstream development projects for both traditional and emerging modalities.Instructor:

Jean-François Hamel, Professor, Academic Researcher and Instructor, Chemical Engineering Department, Massachusetts Institute of Technology

TS14C: Introduction to Biologics Formulation DevelopmentCHI’s Introduction to Biologics Formulation Development training seminar focuses on strategies to plan and execute preformulation and formulation development studies for biologics. Such projects require co-optimization of multiple physical, chemical, and conformational stability attributes under accelerated timelines to ensure rapid delivery of a stabile product to the clinic. The seminar begins with an overview of biophysical and biochemical properties of proteins and protein structure, setting the stage for the concepts and goals at the core of protein formulation. The course then continues with an exploration into the theory and application of the relevant analytical and biophysical techniques that support preformulation and formulation development studies. The primary focus of the seminar is an in-depth discussion of typical formulation development workflows, including statistical analysis and use of DoE. The formulation development section of the course then concludes with examination of real-world case studies. Attendees of the training seminar should leave with a greater understanding of the biologics formulation field, in general, as well as with specific knowledge and strategies for setting up successful formulation development studies in their own labs.Instructor:

Donald E. Kerkow, PhD, Director, Biopharmaceutical Development, KBI Biopharma, Inc.

TS15C: Design of Experiments for Cell and Gene TherapiesDesign of Experiment (DOE) is recognized as the most prominent tool for analyzing the effect of multiple factors on complex unit operations. Cell and gene therapy manufacturing processes contain many classic examples of such operations, and DOE is the best systematic approach for their optimization. This results in improved manufacturing yields, efficiency, cost, consistency and safety. This branch of applied statistics is intimidating, but once explained clearly, surprisingly intuitive and easy to use. This seminar will include specific, practical examples of the application of DOE to optimization cell culture, gene medication and downstream processing unit operations in cell and gene therapy. Attendees will leave with concrete methods and tools enabling them to address their own products’ manufacturing in a sophisticated but practical way.Instructors:

Anthony Davies, PhD, Founder & CEO, Dark Horse Consulting

Andrew Steinsapir, Consultant, Dark Horse Consulting

Jerrod J Denham, Principal & Sr Consultant, Dark Horse Consulting

TRAINING SEMINAR INFORMATION Each CHI Training Seminar offers 1.5 days of instruction with start and stop times for each day shown above and on the Event-at-a-Glance published in the onsite Program & Event Guide. Training Seminars will include morning and afternoon refreshment breaks, as applicable, and lunch will be provided to all registered attendees on the full day of the class.

Each person registered specifically for the Training Seminar will be provided with a hard copy handbook for the seminar in which they are registered. A limited number of additional handbooks will be available for other delegates who wish to attend the seminar, but after these have been distributed, no additional books will be available.

Though CHI encourages track hopping between conference programs, we ask that Training Seminars not be disturbed once they have begun. In the interest of maintaining the highest quality learning environment for Training Seminar attendees, and because seminars are conducted differently than conference programming, we ask that attendees commit to attending the entire program, and not engage in track hopping, as to not disturb the hands-on style instruction being offered to the other participants.



COVER

EVENT AT A GLANCE


POSTERS

CONFERENCE STREAMS

Upstream Processing




Cell Therapy

Gene Therapy

Manufacturing

TRAINING SEMINARS

SHORT COURSES


HOTEL & TRAVEL


Register Online!






SHORT COURSES** Separate registration required

MONDAY, AUGUST 13 | 9:00 - 11:30 AM

SC1A: Optimizing Cell Culture MediaInstructors: Kevin Tan, PhD, Senior Scientist, Irvine ScientificSven Loebrich, PhD, Senior Development Scientist, ImmunoGen, Inc.Ronan O’Kennedy, PhD, Director and Principal Consultant, ROK BioconsultingChentian Zhang, PhD, Engineer I, Bristol-Myers Squibb Co.

SC2A: Data Analytics and Data Management for the Bioprocess LifecycleInstructors: Christoph Herwig, PhD, Professor, Institute of Chemical Environmental and Biological Engineering, Research Division Biochemical Engineering, Vienna University of TechnologyPatrick Sagmeister, PhD, CTO, Exputec GmbHChristopher M. Taylor, MSc, Head, Data Science Consulting, Exputec GmbH

SC5A: Introduction to Host Cell Proteins: Analysis, Characterization, Risks, and RequirementsInstructor: Denise Krawitz, PhD, Principal Consultant, CMC Paradigms LLC

MONDAY, AUGUST 13 | 9:00 AM - 5:30 PM

Full-Day WorkshopW1: Life Science Venture Valuation – Introduction to Company and Product ValuationInstructor: Aitana Peire, PhD, Senior Consultant, Venture Valuation AG

TUESDAY, AUGUST 14 | 6:00 - 8:30 PM | DINNER PROVIDED

SC6B: Protein Aggregation: Mechanism, Characterization and ConsequencesInstructors: Thomas Laue, PhD, Professor Emeritus, Molecular, Cellular and Biomedical Sciences, University of New HampshireMatthew Brown, PhD, Applications Manager, Bioscience, Malvern PANalytical

SC7B: Analytical Strategies for Comparability in Bioprocess DevelopmentInstructor: Christine P. Chan, PhD, Principal Scientist/Technical Lead, Global Manufacturing Science & Technology, Specialty Care Operations, Sanofi

SC8B: Integrated Continuous Biomanufacturing – An Implementation ApproachInstructor: Robert Dream, PE, CPIP, Managing Director, HDR Company Ltd.

THURSDAY, AUGUST 16 | 6:00 - 9:00 PM | DINNER PROVIDED

SC13C: Implementing Phase Appropriate GMP for Manufacturing Biologics and Cell Therapy ProductsInstructor: Trevor Deeks, PhD, QA/QC and GMP Consultant, Deeks Pharmaceutical Consulting Services, LLC

MEDIA PARTNERSWEB PARTNERS:SPONSORING PUBLICATIONS:LEAD SPONSORING PUBLICATIONS:

TM FierceBiotechTHE BIOTECH INDUSTRY’S DAILY MONITOR



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Upstream Processing




Cell Therapy

Gene Therapy

Manufacturing

TRAINING SEMINARS

SHORT COURSES


HOTEL & TRAVEL


Register Online!






Upstream Processing This year’s Upstream Processing stream explores the foundations of protein expression that support manufacturing biological drugs, including cell line development, culturing cells, and issues surrounding bioproduction, such as integrating disposable systems, bioreactor design, and developing small-scale models for scaling up production. Genetic engineering, emerging technologies, and next-gen strategies will be shared as part of the ongoing efforts to ensure quality while achieving manufacturability and bottom-line bioprocess success.

AUGUST 13-14AGENDA Optimizing Cell Culture Technology

AUGUST 15-16AGENDA Bioproduction: Scale, Bioreactors & Disposables

AUGUST 16-17 AGENDA Optimizing Cell Line Development

2018 UPSTREAM PROCESSING TRACKS



COVER

EVENT AT A GLANCE


POSTERS

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Upstream Processing




Cell Therapy

Gene Therapy

Manufacturing

TRAINING SEMINARS

SHORT COURSES


HOTEL & TRAVEL


Register Online!






14TH ANNUAL

Optimizing Cell Culture TechnologyEnhancing Knowledge for Growing Cells

Upstream Processing

MONDAY, AUGUST 13

8:00 am Short Course Registration Open and Morning Coffee

9:00 - 11:30 Recommended Short Course*SC1A: Optimizing Cell Culture Media* Separate registration required; click here for details.

11:30 Main Conference Registration Open

STRATEGIES TO ENHANCE CELL CULTIVATION1:00 pm Chairperson’s Opening RemarksPaula Meleady, PhD, Associate Director, National Institute for Cellular Biotechnology, Dublin City University

1:10 KEYNOTE PRESENTATION: Advancing Cell Culture ProcessesMichael Laird, PhD, Senior Director & Principal Scientist, Process Development, Genentech, Inc.Speed to IND submission is essential in establishing a competitive advantage for a therapeutic product. To further decrease the time and resources required for an IND submission, two strategies have been employed: targeted integration (TI) cell line development (CLD) and pool for tox (PFT). In addition, high titer, robust processes may also be required for commercial production. This presentation will describe our current strategies from early stage to commercial production.

1:45 Quality by Design-Driven Approaches for Reaching Process RobustnessChristoph Herwig, PhD, Professor, Bioprocessing Engineering, Vienna University of Technology (TU Wien), and Founder and Scientific Advisor, Exputec GmbHCell culture processes still lack thorough understanding in reaching higher titers and process robustness using process technological means. This contribution shows different aspects of Quality by Design-driven approaches for limiting amino acids using PAT and automated modelling approaches; model-based experimental designs for targeting maximum information content of the experiment for a given objective, such as media titer optimization or lactate uptake, and scale-down model establishment to analyze the effect of large-scale inhomogeneities.

2:15 Cell Line Sensitivity from Unexpected Sources: A Systematic Investigation of Upstream BioprocessingMaria Choi, Engineer I, Cell Culture Development, BiogenA growth and viability decrease of greater than 50% was observed for a mammalian cell line and was correlated to media preparation but ultimately not to individual media components. Upstream operations were systematically tested, and the root cause was determined to be an inconsistent cleaning procedure. This investigation highlights the importance of upstream operations which may not be ordinarily considered, but which may introduce variability into the cell culture system.

2:45 Refreshment Break

BREAKTHROUGH ANALYTICAL TECHNOLOGIES3:15 Using Real-Time Analysis to Optimize Bioprocesses and Enhance Interactive LearningJean-François Hamel, PhD, Research Engineer & Lab Director, Chemical Engineering, Massachusetts Institute of Technology (MIT)This talk describes how we use real-time analytic tools integrated into process-scale and high-throughput platforms to gain decision-grade information and boost interactive learning during our research. Using these tools generates more actionable information during the experiment, enhances researchers’ capabilities as they can see the statistical analysis and graphs and visualizations which offer a deeper understanding of the processes while the trial is running. We find that users’ experiences are better and researchers’ engagement in the experiment is deeper.

3:45 Raman Microspectroscopic Analysis of Cell Death and DifferentiationJames M. Piret, PhD, Professor, Michael Smith Laboratories, Chemical & Biological Engineering, The University of British ColumbiaRaman spectroscopy is a non-invasive, label-free technology that can analyze cell states based on cellular composition changes. Microspectrometry revealed composition differences over time that distinguished human embryonic stem cells from differentiated pancreatic cell types, such as by increased protein-to-nucleic acid ratios and increased insulin. We have also shown that Raman microspectrometry can distinguish types of cell death. Apoptotic, necrotic or autophagic CHO cells were compared, including sorted cells to determine how early the onset of apoptosis could be detected.

Sponsored by4:15 Continuous In-Line Cell Counting and Infection Kinetics Monitoring, a Case Study Sean Case, Scientist, Upstream Process Development, NovavaxHighlights data from in-Line cell counting compared to off-line counting; infection kinetics at various MOIs (0.1 - 1.2) with fed batch process including two different recombinant protein targets; comparability at 10 L vs 50 or 200 L scales to demonstrate infection kinetics similarity for small scale model qualification (SSMQ).

Sponsored by4:30 An Integrated Approach for Accelerated Cell Line Development and Media Optimization David T. Ho, Senior Scientist II, Research and Development, Irvine ScientificDevelopment of a biotherapeutic protein begins with generating a high-performing stable cell line for manufacturing. Optimization of the culture media is essential to maximize performance and strategies to shorten and streamline this work are in demand. Here we present an accelerated approach integrating which implements a CHO media platform.

4:45 Breakout DiscussionsThis session provides the opportunity to discuss a focused topic with peers from around the world in an open, collegial setting. Select from the list of topics available and join the moderated discussion to share ideas, gain insights, establish collaborations or commiserate about persistent challenges. Then continue the discussion as you head into the lively exhibit hall for information about the latest technologies.



COVER

EVENT AT A GLANCE


POSTERS

CONFERENCE STREAMS

Upstream Processing




Cell Therapy

Gene Therapy

Manufacturing

TRAINING SEMINARS

SHORT COURSES


HOTEL & TRAVEL


Register Online!






14TH ANNUAL


Upstream Processing

5:30 Grand Opening Reception in the Exhibit Hall with Poster Viewing

7:00 End of Day

TUESDAY, AUGUST 14

7:30 am Registration Open and Morning Coffee

CULTURING CHO CELLS7:55 Chairperson’s RemarksBhanu Chandra Mulukutla, PhD, Principal Scientist, Process Development, Pfizer, Inc.

8:00 Proteomic and Phosphoproteomic Characterisation of Growth of Recombinant Chinese Hamster Ovary CellsPaula Meleady, PhD, Associate Director, National Institute for Cellular Biotechnology, Dublin City UniversityDespite phosphorylation being a hugely important post-translational modification, its role in regulating CHO bioprocess-relevant phenotypes has not been studied in detail to date. We describe the phosphoproteomic characterization of CHO cells during growth in culture and in cells subject to temperature shift. Using advanced LC-MS/MS techniques we have characterised > 8000 CHO-specific phosphorylation sites and identified >1000 differentially expressed phosphopeptides in these studies. Phosphoproteins have the potential to be cell engineering targets to improve efficiency of recombinant protein production.

8:30 CHO Growth under Stress Conditions Neil A. McCracken, P.E., MS, Associate Senior Consultant Engineer, Bioproduct Research and Development, Eli Lilly and Company

9:00 Systematic Analysis of Trace Metals on Therapeutic Protein Production in CHO CellsKyle McHugh, PhD, Research Scientist, Upstream Biologics Process Development, Bristol-Myers Squibb Co.A systematic approach was taken to better understand how the levels of specific trace metals may be used to regulate cell culture process parameters and protein quality attributes including N-glycan distribution, charge variant profiles, and protein aggregation. Design of Experiments in high-throughput Tecan liquid handler production runs was used to assess the impact of 12 different metals on 4 proteins of therapeutic interest and confirmed in benchtop bioreactors to generate samples for transcriptomic and metabolomics analysis. Results from the high-throughput screens and follow-up studies will be presented.

Sponsored by9:30 Novel Analytical Solutions for Cell Line DevelopmentCarolanne Doherty, PhD, Manager, Marketing and Sales, ValitacellValitacell is offering the biopharmaceutical industry novel methods to transform the complex manufacturing process that produces bio-therapeutic drugs by making it faster and more cost effective. We aim to fill the gap with our analytical technologies to promote the chance of identifying high-producing stable clones in a reduced time frame.

9:45 Coffee Break in the Exhibit Hall with Poster Viewing

CULTURING CHO CELLS FOR ANTIBODY PRODUCTION10:30 Modulation of Half Antibody and Aggregate Formation in a CHO Cell Line Expressing a Bispecific AntibodyNatalia Gomez, PhD, Principal Scientist, Amgen, Inc.We investigated the modulation of impurity levels in a stable CHO cell line X expressing a bispecific antibody (LC1-HC1+LC2-HC2). In particular, this cell line responded to cell culture temperature by decreasing half antibody formation from 14% to <3% when temperature changed from 36°C to 32.5°C. However, lower temperature also correlated with increased high molecular weight (HMW) species from 4% to 10%. Overall, we identified culture conditions that could alter impurities levels and the overall process yield.

11:00 Challenges in Cell Culture Platform Development of mAb Production with Site-Specific Incorporation of Non-Natural Amino Acid for ADC GenerationWeimin Lin, PhD, Principal Scientist, Cell Culture Development, Ambrx, Inc.We successfully generated a CHO-K1 cell line, stably expressing engineered amber suppressor tRNA and its cognate tRNA synthetase to achieve high production of monoclonal antibodies (mAbs) containing nnAAs. The stable cell lines were further evolved using CRISPR/Cas9 genome editing technology. In this presentation, we will discuss the challenges in cell culture platform development including cell line engineering, systematic DoE-based approaches on optimal chemically defined media and cell culture processes, and, strategies for scale up to clinical and commercial scales

11:30 Engineering Amino Acid Metabolism of CHO Cells towards Reduced Novel Growth Inhibitor Production and Amino Acid PrototrophyBhanu Chandra Mulukutla, PhD, Principal Scientist, Process Development, Pfizer, Inc.In this study, genetic engineering approaches were undertaken to reduce the production of inhibitory byproducts produced from phenylalanine-tyrosine or branched chain amino acid (BCAA) catabolic pathways. Four enzymes in phenylalanine-tyrosine catabolic pathway, originally not expressed in CHO cells, were overexpressed ectopically. Cells overexpressing these enzymes produced lower levels of corresponding byproducts and were able to grow and proliferate in tyrosine-free environment (tyrosine prototrophy). Clones with the KO event produced negligible amounts of inhibitory products associated with BCAA catabolism in fed-batch cultures. As a result of this, these clones reached much higher peak cell densities and produced significantly higher levels of titers in fed-batch cultures.



COVER

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Upstream Processing




Cell Therapy

Gene Therapy

Manufacturing

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14TH ANNUAL


Upstream Processing

Sponsored by12:00 pm Choosing the Right Cell Engineering Technology on CHO Productivity and Therapeutic Development TimelinesJoan Foster, Senior Field Applications Scientist, MaxCyte Working with the manufacturing host cell early in development produces sufficient quantities of candidates for extensive characterization and progression of promising candidates in a cost-efficient manner. Proper means of cell engineering drive rapid, scalable production of proteins in the manufacturing cell background while successfully maintaining flexibility in downstream culture processes. This presentation discusses the high performance and flexibility of MaxCyte’s cell engineering technology and how it impacts therapeutics efficacy and fast-track biotherapeutic development.

Sponsored by12:30 Luncheon Presentation: ExpiCHO, Expi293 and ExpiSf: High Titer Protein Expression for Streamlined Pharmaceutical Development Jonathan Zmuda, PhD, Director, Cell Biology, Thermo Fisher ScientificThe Expi Expression Systems comprise three different cell hosts to provide researchers with unprecedented access to high-titer recombinant proteins in support of all phases of drug/vaccine development. Recent additions to the Expi family of products include GMP banked Expi293 cells, GMP ExpiCHO-S cells (late 2018) and ExpiCHO Stable Production Media to allow for ease of transition from transient to stable ExpiCHO. Here, we introduce and discuss the similarities and differences among the three Expi systems.

Sponsored by1:15 Dessert Refreshment Break in the Exhibit Hall with Poster Viewing

APPLYING METABOLICS TESTING AND INSIGHTS1:55 Chairperson’s RemarksCarolanne Doherty, PhD, Manager, Marketing and Sales, Valitacell

2:00 Driving Commercial Biologics Process Understanding through the Application of Metabolomics TestingAmanda Lewis, PhD, Senior Engineer and Manager, Manufacturing Sciences & Technology, Bristol-Myers Squibb Co.This presentation will explore a methodology for incorporating routine metabolomics testing into a commercial biologics process for continuous improvement and process understanding. LC-MS and NMR data will be presented from a legacy commercial process where metabolomics was used to trend and monitor campaign performance, as well as to establish raw material variability and impact to process performance. Finally, sustainability of this methodology in a commercial biologics space will be addressed.

2:30 13C Metabolic Flux Analysis Identifies Limitations to Increasing Specific Productivity in Fed-Batch and PerfusionNeil Templeton, PhD, Senor Scientist, Upstream Process Development Engineering, Merck Research Laboratories

13C-Metabolic Flux Analysis (13C-MFA) was utilized to compare the stationary phase of a fed-batch process to a perfusion process producing the same product by the same clone. The fed-batch process achieved significantly higher specific productivity, while gross growth rate was approximately 80% greater in the perfusion process. When considering gross growth rate and IgG specific productivity, total protein-specific productivity differed little, offering rationale for the observed central carbon pathway similarities.

3:00 In vivo Quantification of Mitochondrial Shuttle Activities via 13C Flux Analysis in mAB Producing CHO CellsRalf Takors, PhD, Professor, Institute of Biochemical Engineering, University of StuttgartWe have developed a technology that identifies the in vivo mitochondrial shuttle activities of CHO cells during typical production conditions. The compartment-specific 13C analysis distinguishes between cytosol and mitochondrion thereby disclosing the tight coupling of mitochondrial and cytosolic metabolism and underpinning the crucial role of mitochondrion not only as a provider of ATP but also as an essential part of metabolism. Metabolic flux patterns serve as a sound basis for deriving process and metabolic engineering strategies for optimizing mAB productivities further.

Sponsored by3:30 Refreshment Break in the Exhibit Hall with Poster Viewing

NEXT-GEN TECHNIQUES FOR OPTIMAL GROWTH CONDITIONS4:15 Cell Culture Media Characterization and Optimization Using DOE Combined with Multivariate MethodsRonan O’Kennedy, PhD, Consulting Bioprocess Specialist, ROK BioconsultingQuantitative structural activity relationship modelling (QSAR) will be presented as an effective tool to characterize the compositional variability of media containing peptones or hydrolysates. Used in combination with DOE medium optimisation, compositional variability can be linked to cell culture performance. The resulting QSAR based model provides a design space of the effect of compositional variability of commercially available hydrolysates. The model is used to select and optimize peptones/hydrolysates and predict the effects of hydrolysate raw material lot variability on cell culture performance.

4:45 Manipulating Glycan Profile and Application of a High-Throughput Capillary Western Method Using Lectins for DetectionSilvino (Serry) Sousa, MSc, Senior Scientist, Global Protein Sciences, AbbVieI will discuss approaches for modulating N-linked glycosylation of recombinant therapeutic proteins by manipulating media, process and/or genetics of the host cell factory. What is also needed is a rapid, simple, yet protein and titer agnostic method for deriving detailed glycan signature directly and simultaneously from multiple samples of cell culture conditioned medium. I will review methods that we have implemented for rapidly screening for glycan signatures directly from cell culture supernatants.



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14TH ANNUAL


Upstream Processing

5:15 End of Conference

6:00 - 8:30 Recommended Dinner Short Course*SC7B: Analytical Strategies for Comparability in Bioprocess Development* Separate registration required; click here for details.



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8TH ANNUAL

Bioproduction: Scale, Bioreactors & DisposablesMaking It Work

Upstream Processing

WEDNESDAY, AUGUST 15


NEXT-GEN PROCESS DEVELOPMENT8:05 Chairperson’s RemarksThomas Villiger, PhD, Process Expert, Biopharmaceutical Operations, Novartis Pharma SAS

8:15 KEYNOTE PRESENTATION: Next-Generation Biomanufacturing Processes and the Use of Integrated Advanced Process Control ToolsAnne Tolstrup, PhD, Senior Consultant, Bioprocess Technology Consultants, Inc.The availability of biologics drugs keeps increasing with more and more patients being eligible for such therapeutics. To secure safe treatments and adequate drug supply for every patient the biomanufacturing community needs to continue optimization of titers and yields while never compromising on quality. Advanced process control tools for on-line monitoring of controlled process parameters throughout production provides one increasingly employed way of achieving this goal.

9:00 Phase-Appropriate GMP for Biologicals Manufacturing – What Does It Mean and How Should It Be Interpreted?Trevor Deeks, PhD, QA/QC and GMP Consultant, Deeks Pharmaceutical Consulting Services, LLCThe author is the editor of a new book covering Phase Appropriate GMP for Biologicals Manufacturing. There are a huge variety of interpretations of what that means for different types of biological processes at different phases of development and manufacturing from pre-clinical to commercial manufacture. This presentation summarizes the contributions from the many contributors to the book, which describes the current state-of-the-art and industry norms for GMP expectations in Biologicals Manufacturing.

9:30 Application of Machine Learning for Prediction of Product Qualities to Facilitate Decision Making in Manufacturing BioprocessesWei-Chien Hung, PhD, MHS, Process Development Scientist I, Late Stage Upstream Development, Alexion Pharmaceuticals, Inc.Product qualities such as total sialic acid (TSAC) and specific activities are important attributes (CQA) for a therapeutic protein. It has been demonstrated that many factors such as lot-to-lot variability of raw materials and operational variation have potential effects on those product qualities at cell culture fluid (CCF) and harvested cell culture fluid (HCCF). Here we employed machine learning methods to establish a model based on the cell performance from 50 at-scale production bioreactors to predict levels of product qualities. The error rate of prediction is able to be maintained below 10%, therefore becoming useful information for decision making in downstream parameters.


PROCESS INTENSIFICATION10:45 Process Intensification: Current Approaches and Future StrategiesStefan R. Schmidt, PhD, MBA, Head, Operations (COO), BioAtrium AGDisposable equipment is now the industry standard, but the initial replacement of stainless steel instrument is surpassed by innovative strategies to intensify bioprocesses. This has led to the faster generation of cell lines, increased productivity of bioreactors, combination and elimination of process steps in DSP, and finally hugely improved capacity utilization. This presentation gives an overview on current practice and an outlook to future developments.

11:15 Process Enhancement Through Optimized Fed-Batch CulturesSharat Varma, MSc, Engineer II, Cell Culture Process Development, Genentech, Inc.

Sponsored by11:45 High-Density Vero Cell Perfusion Culture in BioBLU 5p Single-Use VesselsAnkita Desai, Bioprocess Field Application Specialist, EppendorfVero cells are anchorage-dependent cells widely used as a platform for viral vaccine production. In this study we cultivated Vero cells in our BioBLU 5p Single-Use Vessels pre-packed with Fibra-Cel and controlled with a BioFlo® 320. We achieved the high Vero cell density of approximately 43 million cells per mL.

Sponsored by12:00 pm Multiplexing Raman Spectroscopy via Seg-Flow Automated Sampling of up to 8 BioreactorsPaul Strand, Senior Application Engineer, Flownamics

12:15 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:00 Session Break

SCALE-UP AND LARGE-SCALE PRODUCTION1:45 Chairperson’s RemarksTrevor Deeks, PhD, QA/QC and GMP Consultant, Deeks Pharmaceutical Consulting Services, LLC

1:50 FEATURED PRESENTATION: Application of Computational Fluid Dynamics for Characterization of Parameters Impacting Scale-Up of Monoclonal Antibody Production ProcessesMichelle LaFond, Senior Director, Bioreactor Scale-Up and Development, Preclinical Manufacturing and Process Development, Regeneron Pharmaceuticals, Inc.Use of both conventional and computational fluid dynamic (CFD) approaches to develop scale-down, pilot-scale models of production bioreactors have resulted in improved process understanding and data-driven transfers of late-stage processes that take the “art” out of scale-up. Combining predictive scale-down models and CFD in our development studies has allowed us to fully characterize ranges of engineering parameters and bioreactor type. These studies yield results that are more informative of how a process will perform at manufacturing scale and promote more robust scale-up. A case study of our approach to scale-up will be discussed.



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8TH ANNUAL


Upstream Processing

2:20 Scale-Up of Continuous Capture ChromatographySrinivas Chollangi, PhD, Senior Scientist, Process Development, Bristol-Myers Squibb Co.Use of multi-column capture chromatography has been demonstrated to offer significant advantages over traditional batch chromatography in regards to improvement in productivity and capacity utilization of the resin. In our recent study, we have compared several multi-column formats (2-column, 4-column and 6-column) and the impact of process parameters such as resin type, feed titer, feed flow rates, % breakthrough and process duration on optimal performance during continuous capture. Upon identifying the optimal conditions, this presentation shows scalability of the process to a pilot-scale purification. Further, we have successfully developed a scale-down model to assess viral clearance across continuous capture chromatography. Insights will be shared on viral clearance across start-up, steady state and shut-down phases as well as effective cleaning strategies to prevent virus carry over between cyclic operations.

2:50 Productivity and Quality Control Optimization of a Large-Scale Multiproduct Biologic FacilityThomas Villiger, PhD, Process Expert, Biopharmaceutical Operations, Novartis Pharma SASThe ever-growing product portfolio of biologics has boosted the demand of more manufacturing capacities worldwide. Given that new facilities involve very high capital expenditures and long times to construct and validate, it is of paramount interest to use the existing manufacturing facilities at its maximum while ensuring product quality at all times. In this presentation, the concepts of different data driven optimizations regarding run rate improvement, productivity increase, batch to batch reproducibility as well as product quality control will be presented.

3:20 MacroBac: New Technologies for Robust and Efficient Large-Scale Production of Recombinant Multiprotein ComplexesJill Fuss, PhD, Research Scientist, Molecular Biophysics and Integrated Bioimaging, Lawrence Berkeley National LaboratoryMacroBac is a multigene baculovirus system that uses ligation-independent cloning for efficient cloning and assembly that is equally well-suited for either single or high-throughput cloning reactions. MacroBac vectors are polypromoter to minimize gene order expression level effects seen in many polycistronic assemblies. Large assemblies are robustly achievable, and we have observed significant increases in expression levels and quality of large, multiprotein complexes over traditional coinfection with multiple, single-gene baculoviruses.

3:50 Refreshment Break in the Exhibit Hall with Poster Viewing

4:45 Plenary Keynote Session (click here for details)

6:25 10th Anniversary Champagne Celebration in the Exhibit Hall with Poster Viewing

7:30 End of Day

THURSDAY, AUGUST 16


OPTIMIZING BIOREACTORS8:25 Chairperson’s RemarksSrinivas Chollangi, PhD, Senior Scientist, Process Development, Bristol-Myers Squibb Co.

8:30 Characterization and Application of Ambr 250 Disposable Bioreactors in Upstream Process DevelopmentPing Xu, PhD, Senior Scientist I, Biologics Development, Global Product Development & Supply, Bristol-Myers Squibb Co.The disposable Advanced Microscale Bioreactors (Ambr 250) system has become a very useful and popular tool, due to the advantages of high throughput, automated control and short turnaround time. In this talk, we will present the characterization of the Ambr 250 to define optimal operation conditions, as well as the assessment of the suitability of Ambr 250 for clone selection. The applications of Ambr 250 in early stage process development for rapid advancement of innovative biologics to First-in-Human (FIH) clinical trials will be also introduced.

9:00 Bridging Clone Screening Platforms with Perfusion Bioreactors for Continuous ManufacturingDavid Busch, PhD, Senior Scientist, Pre-Clinical Development, Merck & Co., Inc.Continuous manufacturing of mammalian biologics offers several potential advantages over batch methods of production. In an effort to optimize a continuous cell culture process, we developed clonal cell lines that were selected for robust performance within a perfusion bioreactor platform. Following clone screening and evaluation using mock perfusion, we evaluated top performing clones in Ambr 15 bioreactors and 2L perfusion bioreactors to bridge clone screening and batch production with a perfusion bioreactor process.

Sponsored by9:30 Latest Advancements in Production Process Intensification for Biopharmaceuticals Alfred Luitjens, Director, Cell Technology, Batavia BiosciencesNovel developments, like the use of fixed-bed bioreactors, allow for extreme process intensification. We are now able to build micro-facilities in which we can perform industrial production. This presentation will give an overview and case study on highly intensified processing to reduce COGs, capital costs, and operating costs.

Sponsored by9:45 Enhancing Bioproduct Knowledge and Bioprocess Understanding with In-Line IR Spectroscopy and Particle Characterization Systems Tyler Gable, PhD, Mettler Toledo AutoChem, Inc.Direct, in-line monitoring of key process parameters in Upstream and Downstream bioprocess operations provide information necessary to monitor and control operations in an effective manner. For example, in situ FTIR spectroscopy provides continuous analysis of bioprocess nutrients, metabolites and products while inline



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8TH ANNUAL


Upstream Processing

particle characterization and image analysis enables direct analysis of particles and aggregates in downstream processing stages. In this presentation, we will provide an overview of the utility of insitu FTIR spectroscopy and inline particle characterization and morphology determination in Bioprocess Development and Biopharmaceutical Manufacturing Stages.


DISPOSABLE SYSTEMS10:45 Application of Alternative Cell Separation Systems for the Harvest of Mammalian Cell Culture Processes in a Fully Disposable Single-Use FacilityDominique T. Monteil, PhD, Scientist II, Cell Culture, Process Science, Boehringer Ingelheim Fremont, Inc.In a fully disposable facility where the use of continuous disk-stack centrifuges are not preferred, harvest processes based on conventional depth filtration become more challenging with increasing single-use bioreactor (SUB) size and higher density culture. Here, several alternative single-use harvest technologies were evaluated. A disposable centrifuge and a range of different synthetic depth filters were tested. Results showed significant improvement in filterability and reduction of depth filter area compared to full traditional depth filtration train.

11:15 Use of Disposable Materials in Bioproduction -- Small Changes May Have a Big ImpactYong Wang, PhD, Senior Director and Head, Process Industrialization, Process Development & Technical Services, Shire, Inc.Single-use systems (SUS) have been widely adapted in biopharmaceutical manufacturing. While SUS offer significant advantages in production over conventional approaches, the polymeric nature of the SUS components poses concerns on chemical leaching into the process stream and the potential impact on product quality. A case study will be presented evaluating the root cause of increased aggregate levels observed in drug substance production and linking the higher aggregate levels with single-use disposable parts employed in downstream processing.

11:45 Equipment Characterization of a New Biologics Facility – Lessons LearnedJose Vallejos, PhD, Manufacturing Scientist, MS&T, AstraZenecaEquipment characterization of new biologics facilities is key to mitigate tech transfer risks. Mass transfer and mixing time studies along with PID tuning of dissolved oxygen and temperature control loops can be time consuming. Poorly planned and executed equipment characterization studies can cause costly delays in a new facility commissioning and start-up of GMP runs. This presentation covers mass transfer studies in rocker bags and stirred stainless-steel bioreactors as well as mixing time studies and PID control tuning (Dissolved Oxygen and Temperature).

12:15 pm Enjoy Lunch on Your Own

1:15 10th Anniversary Cake Break in the Exhibit Hall with Last Chance for Poster Viewing




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10TH ANNUAL

Optimizing Cell Line DevelopmentEnhancing Expression

Upstream Processing

THURSDAY, AUGUST 16

11:30 am Registration Open



CELL LINE DEVELOPMENT STRATEGIES1:55 Chairperson’s RemarksClaus Kristensen, PhD, CEO, GlycoDisplay Aps

2:00 KEYNOTE PRESENTATION: Product Quality Control Strategies during Cell Line DevelopmentZhimei Du, PhD, Director, Cell Line Development, Biologics Process Development & Clinical Manufacturing, Merck Research LabsProduct quality control without compromising productivity has been a major goal in biotherapeutics process development. The challenge is further increased for new modalities using complex and hybrid protein structures. To improve the efficiency in developing a successful production cell line with desired product quality profile, implementation of various near real-time monitoring system at different stage of cell line development would be critical and will be illustrated in this presentation.

2:45 Cell Line Development for Expression of Bispecific and Trispecific MoleculesValentina C. Ciccarone, PhD, Principal Scientist, Cell Line Development, Macrogenics, Inc.

3:15 Improved Recombinant Protein Expression from Mammalian Cells by Manipulating Genes Identified through miRNA and siRNA ScreeningJoseph Shiloach, PhD, Director, Biotechnology Core Laboratory, Intramural Research, NIDDK/NIHFrom a whole-genome RNA interference screen of HEK 293 cells, 10 genes were identified and validated by their effect on protein expression. From these genes, OAZ1 was selected for further investigation because its silencing improved protein production without affecting cell viability and because its role in polyamine metabolism is known. Another protein enhancing gene, HIPK1, was identified by utilizing both miRNA and siRNA screening. The gene identification process and the creation, the growth, and production properties of the engineered cells will be described.

Sponsored by3:45 A Novel Approach to Cell Line Selection Using RNA Ted Eveleth, CEO, HocusLocus, Inc.Our novel approach to cell line selection replaces marker proteins, like DHFR and GS, with an siRNA contained within an intron in the gene of interest transcript. Subsequent selection can be performed by transfecting mRNA and then using either FACS or magnetic-based isolation to produce a pool.


NEXT-GENERATION CELL LINE ENGINEERING4:15 Programmed Gene Regulatory Networks for Cell Line EngineeringRon Weiss, PhD, Professor, Biological Engineering, Massachusetts Institute of Technology (MIT)Synthetic biology is revolutionizing how we conceptualize and approach the engineering of biological systems. Recent advances in the field are allowing us to expand beyond the construction and analysis of small gene networks towards the implementation of complex multicellular systems with a variety of applications. In this talk, I will describe our integrated computational/experimental approach to engineering complex behavior in a variety of cells, with a focus on mammalian cells.

4:45 Utilizing RNA Epigenetic Modifications to Enhance Transgene ExpressionNiall Barron, PhD, Professor, Biochemical Engineering, National Institute for Bioprocessing Research and Training (NIBRT), University College DublinOne modified base that has generated considerable interest recently is N6-methyladenosine (m6A), which is one of the most common epigenetic modifications found on RNA. While much effort has been dedicated to elucidating the role of m6A, there remains a lot of questions around its true biological role. Our interest has focused on its potential as an enhancer of mRNA translation and how this mechanism might be applied to improving recombinant protein expression systems.

5:15 End of Day

6:00 - 9:00 Recommended Dinner Short Course*SC13C: Implementing Phase Appropriate GMP for Manufacturing Biologics and Cell Therapy Products* Separate registration required; click here for details.

FRIDAY, AUGUST 17


DEVELOPING CHO-BASED CELL LINES8:25 Chairperson’s RemarksNiall Barron, PhD, Professor, Biochemical Engineering, National Institute for Bioprocessing Research and Training (NIBRT), University College Dublin

8:30 FEATURED PRESENTATION: Robust and Flexible Approaches towards High Quality Cell LinesVolker Sandig, PhD, CSO, Probiogen AGIn this talk, we will discuss how the CHO starter cell, vector, culture media and even selection principles can drive performance of a cell line development platform and show implications for glycoproteins as well as bispecific antibodies. We will also address challenges to verify single cell origin of the final cell line and describe a novel approach for gentle cloning with embedded validation co-developed with Automated Lab Systems (ALS).



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10TH ANNUAL


Upstream Processing

9:00 Optimization of a Stable CHO-K1-Based Cell Line for FAP-DR5 2+2 CrossMab Antibody in Order to Increase Product Quality and YieldStefan Seeber PhD, Principal Scientist, Molecule & Cell Line Development, Roche Innovation Center PenzbergWe have generated a recombinant, stable CHO-K1-based cell line for a novel tetravalent FAP-DR5 (2+2 type CrossMab) antibody, which effectively triggers FAP-dependent, avidity-driven DR5 hyperclustering and tumor cell apoptosis. The application of our cell line development platform comprising semi-automation and a diligent cell line selection strategy resulted in final cell clones, which showed a stable product profile and high yield, but side peaks which referred to limitations in crossed light chain expression. In the talk, we describe the strategy and execution of targeted cell line optimization leading to a final cell line with improved quality and yield.

9:30 Evaluating CRISPR-Cpf1 as a Genome Editing Technology in Chinese Hamster Ovary (CHO) CellsZhuo Cheng, PhD, Research Scientist, Molecular and Cell Biology, Bioprocess R&D, Eli Lilly and CompanyCpf1 has been reported to be highly specific and efficient in gene editing, at levels approaching that of the high fidelity Cas9 in human cells. This study demonstrates that CRISPR/Cpf1 offers a viable alternative to the commonly-leveraged CHO cell gene-editing tool ZFN. In a study, Cpf1 produced significantly more biallelic genetic knockouts than ZFN. Use of CRISPR/Cpf1 was successfully integrated into the cell line generation process with no negative impacts on timeline, screen size or production cell line productivity.

10:00 Networking Coffee Break

DEVELOPING CHO-BASED CELL LINES10:30 Unlocking the Mysteries of CHO Cells Using Transcriptomic AnalysisPhilip Probert, PhD, Senior Upsteam Scientist, Biologics, The Centre for Process Innovation (CPI)RNA Seq analysis using the Ion Torrent S5 instrument was used to characterise the gene expression profile of a number of CHO cell variants edited using CRISPR/Cas9 and grown under industrially relevant conditions using an ambr 250 bioreactor system. Our results demonstrate that RNA Seq analysis provides an insight into the intracellular dynamics of mammalian cells during bioprocessing and could provide opportunities for cell engineering by identifying potential gene targets to improve performance.

11:00 Optimization of Expression Vector Construction for Clinical Cell Line DevelopmentYizhou Zhou, PhD, Scientist II, Cell Culture Development, BiogenOptimal protein expression in stable CHO cells often requires optimization of the expression vectors. To streamline the transition from Research to Development, we have evaluated effects of different codon editing strategies and signal peptide choices on stable cell line development. In addition, cloning strategies were also optimized to speed up the vector construction process.

11:30 Platform Automation and High Throughput Screening to Improve Monoclonal Antibody Production in CHO CellsDaniel Kordella, MSc, Associate Scientist, Cell Line Development, NIH/NIAIDOur group has been working towards incorporation of two large/high throughput technologies in our cell line development process. The first is a large-scale liquid handler capable of automated expansion of cell lines at every stage. The second is incorporation of a HTP screening platform capable of using multiple criteria to identify candidate cell lines and mark them for expansion. The two platforms are integrated in such a way that data from screening is used to rapidly mark cell lines for expansion, which is then carried out automatically by the liquid handling platform.

12:00 pm Sponsored Presentation (Opportunity Available)


1:15 Session Break

BREAKTHROUGH TECHNOLOGIES TO IMPROVE CELL LINE DEVELOPMENT1:25 Chairperson’s RemarksZhuo Cheng, PhD, Research Scientist, Molecular and Cell Biology, Bioprocess R&D, Eli Lilly and Company

1:30 Automate Research Cell Line Generation with I-Cell TechnologyLeyu Wang, PhD, Senior Scientist II and Project Leader, Global Protein Sciences – Biologics, AbbVie Bioresearch CenterEngineering cell lines with surface antigen expression is essential for the discovery of new biologics targeting receptors. We developed an automated system iCell (Isolated Cell Culture) with the use of CellRaft arrays for culturing thousands of single cells in a small footprint to speed up single cell cloning and generate high quality cell lines with minimal manual intervention. Proof-of-concept studies and efforts to automate the ICell technology will be presented.

2:00 Optimizing Biologics by Cell-Based GlycoengineeringClaus Kristensen, PhD, CEO, GlycoDisplay ApsDr. Kristensen will discuss his group’s cell-based glycoengineering platform that displays different glycan structures on biologic candidates for optimization. Many protein researchers do not know which glycoform is optimal for a particular protein, therefore, Dr. Kristensen’s group develops a series of glycovariants of a protein for screening in established assays. The presentation will introduce their cell-based platform and how they apply their technology to optimize antibodies and other proteins.



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10TH ANNUAL


Upstream Processing

2:30 Leaping-in to Cell Line Development: An Evaluation of ATUM’s Leap-in Transposase SystemNeal Schilling, PhD, Senior Scientist, Process Sciences, CHO Cell Line Development, AbbVie Bioresearch CenterDr. Schilling will show data from an in-house evaluation of ATUM’s Leap-In® Transposase system. A cell line development campaign for a model mAb, from bulk pool generation through cloning and stability, will be presented.

3:00 Higher Throughput Cell Line Development Using the VIPSThomas Kelly, Scientist, Cell & Developability Sciences, Janssen R&DStandard limiting dilution cloning for CLD typically requires seeding densities of 0.5 cells/well in many 96-well plates to obtain enough clones for further evaluation. This results in mostly empty wells being plated, imaged, and eventually consolidated. Solentim’s Verified in situ Plate Seeding (VIPS) system was recently released to the marketplace. It allows for automated seeding of cells at a target of 1 cell/well with imaging. This allows for fewer 96-well plates seeded to screen the same amount of clones. This saves time in plating, imaging, consolidating clones, and titering.

3:30 Close of Conference



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Downstream Processing Downstream processing is an area ripe for technology innovations. The need to meet high upstream titers and the demand for lower cost are pushing companies to look at more creative strategies in purification and recovery. The weeklong Downstream Processing stream will highlight technologies such as continuous/semi-continuous processing, new affinity resins, membrane chromatography, high throughput and miniaturization technologies, PAT and process modeling, as well as novel tools to detect and control viruses and pathogens, including host cell proteins, aggregates, leachables and more.

AUGUST 13-14AGENDA Continuous Processing in Biopharm Manufacturing

AUGUST 15-16AGENDA Advances in Purification and Recovery

AUGUST 16-17

AGENDA Detection, Characterization and Control of Impurities in Biologics

2018 DOWNSTREAM PROCESSING TRACKS



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Manufacturing

4TH ANNUAL

Continuous Processing in Biopharm ManufacturingEnabling Technologies & Creative Approaches


MONDAY, AUGUST 13


9:00 - 11:30 Recommended Short Course*SC2A: Data Analytics and Data Management for the Bioprocess Lifecycle* Separate registration required; click here for details.


1:00 pm Chairperson’s Opening RemarksAndrew Sinclair, President & Founder, Biopharm Services Ltd.

1:10 KEYNOTE PRESENTATION: Continuous Chromatography and Process Integration for BiotherapeuticsThomas Mueller-Spaeth, PhD, Senior Scientist, Institute for Chemical and Bioengineering, ETH ZurichContinuous chromatography is a major facilitator of cost and process time savings in biotherapeutics production. Further savings and product quality improvements result from integration with continuous upstream and minimization of hold steps.

NEW TECHNOLOGIES AND APPROACHES1:45 Optimized Process Control Using Biocapacitance Probes for Large Scale Continuous ManufacturingJacob Jensen, Senior Manager, Manufacturing Sciences, Program Management, Biogen DenmarkOptimizing process controls to maximize your manufacturing reliability is significantly increased when applying PAT tools to your process. Capacitance and Raman are great examples of inline control which can serve the purpose of tight process control enabling consistent product quality output.

2:15 Development of Continuous Capture Process of Antibodies Using Different Protein A Resins: Loading Strategy, Process Design and Other ConsiderationsXuezhen Connie Kang, PhD, Principal Scientist, Continuous Manufacturing Skill Center, SanofiSanofi is currently developing an integrated and continuous bio-manufacturing (ICB) platform for the universal production of protein therapeutics (antibodies, enzymes, etc.). This presentation will focus on continuous capture of antibodies on the Protein A column. Different resins with a variety of cost, binding capacity, and flow characteristics are evaluated under a series of operation conditions. The data were used to develop continuous capture process of antibody therapeutics. Continuous chromatography process design strategies are discussed.


3:15 Synthetic Peptide Ligands for Improved Clearance of CHO Host Cell ProteinStefano Menegatti, PhD, Professor, Chemical and Biomolecular Engineering, North Carolina State UniversityTraditional HCP clearance relies on a combination of bind-and-elute or flow-through steps with broad specificity. While effective at attaining high MAb purity, this approach does not address “difficult” species such as MAb-bound or enzymatically active HCPs. To address these issues, we have developed HCP-binding peptide ligands for use as either pre-Protein A or polishing steps to enhance HCP removal in flow-through mode.

3:45 PAT Systems: Leveraging Historical Batch Process Data to Stimulate Continuous Process DevelopmentNicholas Trunfio, PhD, ORISE Fellow, US Food & Drug Administration Multivariate calibration models facilitate the real-time monitoring of cell culture nutrients and metabolic byproducts, but they require robust training data sets to be accurate and reproducible. This work demonstrates that when the right preprocessing techniques are applied to historical batch data, the resulting models can be used to estimate the same parameters in continuous cell culture processes while more relevant training data sets are acquired.

4:15 FEATURED PRESENTATION: Regulatory Research to Support PAT Implementation in Continuous BiomanufacturingErica J. Fratz-Berilla, PhD, Staff Fellow, Office of Biotechnology Products, US Food and Drug AdministrationThe Guidance for Industry on PAT drafted in 2004 has been one of the most influential guidances in bioprocessing as it has offered a context on which to build innovative pharmaceutical development, manufacturing, and quality assurance. Regulatory research is essential to support regulatory decisions, policy development, FDA guidance documents, and reviewer training. Several research examples in support of PAT implementation in upstream continuous biomanufacturing will be presented.



7:00 End of Day



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Manufacturing

4TH ANNUAL



TUESDAY, AUGUST 14


INTEGRATED CONTINUOUS BIOPROCESSING7:55 Chairperson’s RemarksStefano Menegatti, PhD, Professor, Chemical and Biomolecular Engineering, North Carolina State University

8:00 FEATURED PRESENTATION: Innovative Manufacturing Strategies - What Are the Economic Drivers?Andrew Sinclair, President & Founder, Biopharm Services Ltd.In this talk, we use advanced whole bioprocess models to understand the drivers and impact of these different modalities. The talk will explore upstream approaches such as concentrated N-1 seed, perfusion and concentrated fed batch, and in the downstream arena, straight through processing, the opportunities for continuous (a single operation vs. the whole process) and the potential for new approaches such as membrane technology. The outcomes of modelling will provide insights into the benefits and the issues that need to be addressed for a successful financial outcome.

8:30 Building a Fully Integrated Continuous Bioprocessing cGMP Plant: Promises It Holds and Lessons Learned from ItSamir Varma, General Manager, Head of Manufacturing, Enzene BiosciencesConnecting the perfusion bioreactor to the continuous chromatography system creates a continuous flow of drug substance and promises to reduce Capex and COGs. Automation and PAT tools are essential. Enzene Biosciences is building a cGMP plant that would have a fully integrated continuous bioprocess. We have already completed a proof-of-concept studies in pilot scale (50L). In this talk, I will share our experience of this project with the industry.

9:00 Design Principles and Performance Data of an Integrated Continuous Antibody Downstream ProcessMartin Poggel, PhD, Manager, Biotech Projects, Bayer AGThe envisioned production concept for therapeutic proteins relies on continuous processing in combination with a modular, single use and closed processing design. Following these principle rules the setup will contribute to a higher degree in flexibility and capacity in comparison to a standard batch-wise operated facility. The presented concept is able to handle the demand stretch in clinical and commercial manufacturing from a few kg to large quantities in the X00 kg per year amount at constant product quality.

Sponsored by9:30 Virus Clearance in Continuous Multi Column ChromatographyMark Schofield, Senior Research & Development Manager, Pall Biotech

Continuous bioprocessing is increasingly seen as a means to decrease the cost and increase the quality of biotherapeutics. A key regulatory requirement is the demonstration of virus safety. What impact does switching to continuous chromatography have on the virus clearance of the Protein A step?


FROM BATCH TO CONTINUOUS10:30 Cell-Controlled Perfusion and Its Applicability to Hybrid Perfusion Fed-Batch ProcessesMeredith Curran, MSc, Scientist, Culture Process Development, PfizerA method by which cells effectively control their own rate of perfusion to meet optimal nutritional requirements while controlling lactate accumulation has been applied to the production process development of a monoclonal antibody. By utilizing the cell-controlled perfusion method for a short duration, followed by conventional fed-batch operation with highly concentrated feeds, significant improvements in overall productivity were achieved.

11:00 Rapid Conversion of a Low Productivity Fed Batch to a High Productivity Perfusion Process to Support Fully Continuous ManufacturingWilliam Nicholas Napoli, Scientist, Bioprocess Technology and Expression, Bioprocess Development, Merck & Co., Inc.

11:30 Next-Generation Biopharmaceutical Downstream Process - An EU Horizon 2020 Funded Research and Innovation ProgrammeMichael W. Phillips, PhD, Director, Next Generation Bioprocessing R&D, MilliporeSigmaNextBioPharmDSP is an EU funded research program focused on implementing a fully integrated, continuous downstream manufacturing platform that leverages single-use technologies. Evaluation and optimization of various continuous harvest, capture, and polishing technologies were conducted at small-scale, with final selection based on a holistic process to meet purification and yield targets while minimizing overall economics. Leveraging advanced analytical tools at various points within the process, pilot-scale proof-of-concept trials of the integrated continuous process were completed.

Sponsored by12:00 pm Developing a New Protein A Resin Toolbox to Increase Commercial ProductivityPatrick Gilbert, PhD, Research & Development Manager, Agarose, Purolite Life SciencesA new methodology of resin manufacturing has been utilised to make a high capacity protein A resin with enhanced alkaline stability. Advantages of the new production method will be discussed as well as commercial economics at process scale.

Sponsored by12:15 Paving the Way to Integrated Continuous Bioprocessing: In-line PAT for Steady-State Purification with the CCTC PlatformDmitriy Fedorenko, Associate Director, Process Sciences, ChromaTan



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4TH ANNUAL



CCTC is a column-free purification platform with unique opportunities for PAT. CCTC’s steady-state operation is a natural fit for simplified PAT and integrated processing. New data on CCTC integration of proA with polishing steps using real-time PAT for mAb yield and aggregate shows system productivities > 100 g /L /hr.

Sponsored by12:30 Luncheon Presentation: Maximizing Process Intensification and Cost-Savings in Next-Generation Monoclonal Antibody Production Michael Felo, Director, Downstream Process Integration, MilliporeSigma


CONTINUOUS PROCESSES FOR NOVEL BIOTHERAPEUTICS1:55 Chairperson’s Remarks

Samir Varma, General Manager, Head, Manufacturing, Enzene Biosciences

2:00 Continuous Processes for Glyco-Optimized or Difficult-to-Express ProteinsVicky Goralczyk, PhD, Scientist USP, Glycotope GmbHEven though productivity for CHO systems has remarkedly improved over the last years, some biopharmaceuticals like bispecific constructs or complex glycoproteins remain very challenging. With fed-batch being often the cultivation system of choice, issues of low quality or reduced productivity may arise. With Glycotope’s more than a decade of perfusion expertise, I present case study data from upstream perfusion development for the human GlycoExpress cell line GEX, which overcomes productivity and product quality limitations.

2:30 Development of a Perfusion Cell Culture System for the Manufacturing of Allogenic Exosome-Based BiotherapeuticsAndrew Grube, Senior Research Associate, Upstream Process Development, Codiak BiosciencesAs efforts accelerate to translate exosome biology into new medicines, clear technology gaps have emerged between the current state of the art for producing these vesicles, and the capabilities necessary to support large scale clinical and commercial manufacturing. To this end, Codiak BioSciences has leveraged cutting edge bioprocessing methods developed through decades of recombinant protein manufacturing to create a scalable exosome production platform based on an immortalized human cell line growing in suspension and CD media.

3:00 Control of Protein Glycosylation in a Continuous Bioreactor ProcessRustin Shenkman, PhD, Senior Development Engineer, BDS Process Development, Shire


4:15 Continuous Chromatography for Oncolytic Virus PurificationRicardo J.S. Silva, PhD, Scientist, Animal Cell Technology Unit, iBET Instituto de Biologia Experimental TecnologicaOncolytic virus manufacturing scale can range from 5L in research and development up to 50L for clinical studies and reach around 500L in commercial scale. Continuous chromatography offers a transversal solution to match the scale of the manufacturing plant to any of the throughputs, easing technology transfer and lowering investment costs.

4:45 Continuous Downstream Processing of Non-mAb BiologicsJi Zheng, PhD, Associate Director, Biologics Development and Manufacturing, CelgeneThe increasing production volumes of biomolecules and a rising cost pressure from the market has engendered a strong interest in the downstream chromatographic purification steps. Continuous processing could potentially reduce operational costs and has been applied in industries for monoclonal antibody (mAb) manufacturing. However, continuous downstream processing for non-mAb biologics is still challenging. In this presentation, the strategies and technologies that can be implemented into continuous downstream processing for non-mAb biologics will be discussed.


6:00 - 8:30 Recommended Dinner Short Course*SC8B: Integrated Continuous Biomanufacturing - An Implementation Approach* Separate registration required; click here for details.



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4TH ANNUAL

Advances in Purification & RecoveryNew Materials, Technologies and Processes for Novel Products




PROCESS INTENSIFICATION - MODELLING, HIGH THROUGHPUT METHODS AND PROCESS CONTROLS8:05 Chairperson’s RemarksDavid W. Wood, PhD, Professor, Chemical and Biomolecular Engineering, Ohio State University

8:15 KEYNOTE PRESENTATION: Advanced Modelling of Process Chromatography Combining CFD with Mass Transfer ModelsAlois Jungbauer, PhD, Professor, Biotechnology, University of Natural Resources and Life Sciences, Vienna and Austrian Centre of Industrial BiotechnologyFor a complete description of process chromatography, the packing of the column including the extra column space and the mass transfer must be considered. This is only possible when the convective flow is described by CFD and the diffusion into the pores by mass transfer models. An example will be given for scale up from 1 ml column to 65 L columns. Methods to implement it in process development will be discussed.

9:00 Feedback Control System for mAb Downstream PurificationLizy Kaiyan Tang, MSc, Senior Scientist, MMD, BVA, PAT, MerckA feedback control system for monoclonal anitbody downstream purification established by liquid chromatography the analyzer, prep scale chromatography the purification system and PAT software the brain. This talk will illustrate how this system enables real time product quality control as well as its extended applications.

9:30 Implementation of Design Space Strategy to Control Product Quality During Large Scale Purification of Therapeutic ProteinsIonela Iliescu, Msc, Scientist I, Technical Development, BiogenDesign Space/QbD strategy offers great flexibility in dealing with adsorbent lot variability in commercial scale manufacturing. The information contained in the chromatography certificate of analysis may not be sufficient to evaluate adsorbent lot performance for a specific process and product separation. This presentation will describe the development of a chromatography step with a control strategy which accounts for adsorbent lot to lot variability, the regulatory considerations, and the activities required to implement it in manufacturing.


10:45 Modular Buffer Mixing for Automated Membrane Protein Purification Using AkTA AvantJonas Lee, PhD, Scientist, Protein Technologies, AmgenTransmembrane proteins are important targets in therapeutics discovery. Yet they are difficult to express and purify due to its exposed hydrophobic regions requiring

complex buffer formulation that needs to be screened. I will present various automated high-throughput methods to screen for best solubilizing conditions for membrane proteins followed by modular buffer mixing capability of AkTA Avant to purify multiple membrane proteins in different buffer conditions automatically.

11:15 NEW: Viral Filtration DoE Screening Study for a Biologic Using A Model BacteriophageZhen Tong, PhD, Scientist, Biologics Manufacturing Science & Technology, Bristol-Myers SquibbIn this study, a full-factorial viral filtration (VF) screening DoE study for a biologic product was performed to determine the effects of differential pressure, load protein concentration, load volume to surface area and pause on viral clearance capacity and filtration flux. Facility fit requirements were considered to set the process ranges for the study. A model bacteriophage was used as a model virus for a parvovirus. This bacteriophage possesses very similar particle size and physio-chemical properties as a parvovirus but can be purified at higher titer and requires a significant shorter infectivity assay time, which makes it an optimal virus for high-throughput DoE studies. From these results an empirical model was derived and operational process ranges were identified for use in a subsequent viral clearance validation study with a full panel of model viruses.

Sponsored by11:45 New Depletion Technology for Mid-Stream and Downstream Purification Operation Fabien Rousset, PhD, Head, Bioseparations, Business Unit, DAICEL/Chiral Technologies EuropeDue to increased levels of process-related impurities, midstream operations are limited and challenging. Depletion performances for HCP, DNA, and other target mABs are improved with this new SU flexible technology thanks to several removal mechanisms. Classic technologies can be combined into a single-step unit, which operates pre-protein A. This enhances process safety and overall purification platform performances.

12:15 pm Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:00 Session Break

NON-PLATFORM DOWNSTREAM PROCESSES FOR EMERGING PRODUCTS1:45 Chairperson’s RemarksAlois Jungbauer, PhD, Professor, Biotechnology, University of Natural Resources and Life Sciences, Vienna and Austrian Centre of Industrial Biotechnology

1:50 ProTIA: Challenges with Isolation and Purification of a Novel, XTENylated Bispecific T Cell Engager TherapeuticUlrich Ernst, COO & Senior Vice President, Technical Operations, AMunix Operating, Inc.Amunix has created a bispecific T cell engager platform in a pro-drug delivery format



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4TH ANNUAL



that overcomes current, on-target/off-tumor challenges related to conventional antibody constructs. Challenges to manufacturing and scale up of the molecule have been successfully overcome by a priori selection and incorporation of affinity domains into the molecule design. Data and results are presented demonstrating how these design features have been successfully utilized to enable efficient high throughput screening, process development and scale up to GMP requirements.

2:20 Purification Methods for Higher Throughput Generation of Bispecific AntibodiesAdam Zwolak, PhD, Senior Scientist, Biologics Discovery, Janssen R&DBsAbs comprised of mutant and wild-type IgG subunits can be efficiently separated from contaminating parental mAbs by differential protein A elution starting from either a) purified parental mAbs, b) in-supernatant crossed parental mAbs, or c) co-transfected mAbs. We show that the Q311R mutation confers enhanced FcRn interaction in vitro, and Abs harboring either the Q311R or TLQ mutations have serum half-lives as long as wild-type human IgG1. Together, the results lead to a method for high-throughput generation of BsAbs suitable for in vivo studies.

2:50 A Practical Self-Cleaving Tag Platform for Initial Capture of Complex GlycoproteinsDavid W. Wood, PhD, Professor, Chemical and Biomolecular Engineering, Ohio State UniversityWe present our latest work on a split-intein system for the capture and purification of complex glycoproteins. This system is based on a capture resin, which immobilizes the tagged target while generating a cleavage-competent complex to release the tagless and traceless target after purification. This presentation will detail new information on the performance of the system with various targets, and provides guidance on predicting and enhancing cleavage performance in laboratory and manufacturing settings.

3:20 Challenges in Purifying Influenza Virus-Like ParticlesCristina Peixoto, Head, Downstream Process, Animal Cell Technology, iBET Instituto de Biologia Experimental TecnologicaHere we describe the establishment of an improved DSP unit train platform, adapted from virus particles to influenza VLPs, using pseudo-affinity sulfated cellulose membrane adsorbers (SCMA). Using this SCMA platform for influenza virus particles purification, we were able to speed up the process by decreasing the number of DSP steps, to improve the scale-up and to reduce costs due to the removal of other chromatographic steps.




7:30 End of Day

THURSDAY, AUGUST 16


NEW MATERIALS, TECHNOLOGIES AND APPROACHES8:25 Chairperson’s RemarksAndrew Kim, PhD, Senior Scientist, Global MSAT, Sanofi

8:30 A Universal Platform for Protein Purification (UPPP) by Liquid ChromatographyXindu Geng, PhD, Professor, Separation Science and Technology, Northwest University, Suzhou, ChinaA universal platform for protein purification (UPPP) by multiple dimensional liquid chromatography (mD-LC) which is suitable for ultrafast purification with simultaneously high resolution of proteins is established. All assisted operations in mD-LC, such as desalting, buffer exchange, re-concentration of target proteins, etc., can now be done in a closed and positive pressure system. Target proteins can be quantitatively transferred from previous separation mode to subsequent mode, greatly increasing mass recovery, separation speed, reproducibility, and sample loading.

9:00 Continuous Virus Inactivation Process Using a Novel Packed-Bed ReactorDuarte L. Martins, PhD Student, Lab of Protein Technology and Downstream Processing, Dept of Biotechnology, University of Natural Resources and Life Sciences ViennaContinuous virus inactivation has been overlooked while the biopharm industry moves towards continuous integrated processing. A novel continuous virus inactivation setup, which has several advantages over other approaches, will be presented. This talk will focus on ta case study in which solvent/detergent treatment was employed for continuous inactivation of two-industry-standard virus models (X-MuLV and BVDV). Control experiments will be presented showing that the continuous setup is as effective as the batch incubation.

Sponsored by9:30 LigaTrap® Technologies: Advancements in IgM Purification Tee Bordelon, PhD, CSO, LigaTrap TechnologiesLigaTrap® Technologies has developed a novel, proprietary portfolio of peptoid and peptide-based affinity ligands, specific for the purification of monoclonal and polyclonal immunoglobulins from various species. In addition to IgG, IgA, and IgY purification products, we have developed an IgM purification resin that is superior to all other IgM purification products on the market. Binding capacities of the LigaTrap® IgM purification resin exceeds 15mg IgM per ml of resin and offers excellent selectivity and purity.




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10:45 Enhanced Affinity Capacity via Novel Utilization of Hydrophobic InteractionsKeith Selvitelli, Senior Associate Scientist, Process Biochemistry, BiogenProtein-ligand binding/interaction presents a delicate balance between charges on the ligand and the conserved residues on the molecule. To examine the effects and dependence of salt type and salt concentration on capacity and recovery from novel/custom affinity chromatography step, protein-ligand binding kinetics were studied. Preliminary results provided evidence for enhanced retention resulting in better resin bed capacity utilization, higher loading capacities, improved yields, smaller bed volumes and a general reduction in cost of goods.

11:15 High Concentration Ultrafiltration Challenges in Manufacturing PlatformMahsa Hadidi, PhD, Process Engineer III, Global MSAT Process Sciences, Sanofi GenzymeUltrafiltration is the most commonly used method for concentration and diafiltration of nearly all recombinant therapeutic proteins. However, there are unique challenges in applying this technique to obtain very high target concentration that is needed for monoclonal antibody (mAb) formulations. This work is a case study discussing challenges in a manufacturing platform and the approach towards easy-to-implement resolutions to overcome these difficulties.

11:45 Mechanisms of mAb Viral Filtration Improvement by Nylon PrefiltersVitali Stanevich, PhD, Process Development Scientist, Janssen PharmaceuticalsWe found that nylon membranes significantly improved mass throughput during mAb viral filtration step in comparison to target conditions. The Definitive Screening Design (DSD) experiment showed ionic nature of impurity removal and protection. In particular, nylon prefilters showed themselves as weak-anion exchangers, where mass-throughput directly correlated with pH and reversely with conductivity. Our findings can contribute to optimization of cost, increased throughput, and reduction of development time when using a nylon membrane as a prefilter.






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INAUGURAL Detection, Characterization and Control of Impurities in BiologicsHot Topics, Emerging Contaminants and Impurities, Case Studies and New Technologies


THURSDAY, AUGUST 16




REGULATORY PERSPECTIVE, REFERENCE STANDARDS AND OTHER CONSIDERATIONS1:55 Chairperson’s RemarksDiane Paskiet, MS, Director of Scientific Affairs, Scientific Affairs and Technical Services, West Pharmaceutical Services

KEYNOTE PRESENTATIONS2:00 Control of Impurities for Novel Biologic and Biosimilars from Regulatory PerspectiveAudrey Jia, PhD, Principal Consultant, Regulatory Affairs, DataRevive LLC, Ex-FDA, Office of BiotechnologyBiological products contain product-related and process-related impurities that are derived from the expression system and the manufacturing process. This talk focuses on discussions during the clinical development of the biological products, how impurities are evaluated for its quality and related safety and efficacy, and how the impurities are controlled for novel biologic and biosimilar products. Case studies for impact of impurities and regulatory considerations are presented.

2:45 Approaches to Monitoring Impurities Using USP StandardsKevin Carrick, PhD, Director, Science & Standards, Global Biologics, United States Pharmacopeia (USP)Impurities must be monitored and controlled to ensure the quality of the drug substance and drug product. Impurities range from process-related, such as host cell DNA, host cell proteins and particulates, to product-related, such as degradants, precursors and aggregates. This presentation will outline approaches found in the USP for monitoring impurities from process-related to product-related.

3:15 Continuing Advances in Risk Mitigation in the Use of Fetal Bovine and Other SeraRosemary J. Versteegen, PhD, CEO, International Serum Industry AssociationThis presentation will focus on the mitigation of risk in the use of animal sera in cell culture. The talk will review and provide new data on programs such as traceability certification, geographic testing of origin, age determination of source animals, and gamma irradiation of animal sera.

Sponsored by3:45 What Happens When You Combine Traceability with ScienceKate Linterman, Business Development, OritainForensic science can be applied to products we use every day to trace where they have come from. From the food we eat to the clothes we wear and the raw ingredients that go into our pharmaceuticals. This presentation explains the technology and its applications through real in-market examples.


4:15 New Reference Standards for Monitoring Proteinaceous Particles in BiotherapeuticsSrivalli Telikepalli, PhD, Research Chemist, Biomolecular Measurement Division, National Institute of Standards and TechnologySubvisible and visible particle standards are being developed using an ethylene tetrafluoroethylene polymer, which better mimics the morphology and optical properties of proteinaceous particles. These particles can be used to standardize subvisible particle measurements and to better understand various instrument biases. The development and various applications of this standard, along with the current efforts to standardize the visual inspection methodology will be discussed.

4:45 Sources of Particles in Biologic Products: Considering Assessment of Packaging MaterialsDiane Paskiet, MS, Director of Scientific Affairs, Scientific Affairs and Technical Services, West Pharmaceutical ServicesIt is critical to understand the types and sources of particulates in biologic products and how their presence may affect product quality and patient safety. The linkage between particles, biologic formulation and packaging development involves understanding contributions from all sources. This presentation will provide insight into measurements of particles originating from packaging components, comparison of data from various technologies and current standards.

5:15 End of Day

6:00 - 9:00 Recommended Dinner Short Course*SC11C: Development of Innovative and Competitive Biologic Formulations in Complex Patent Landscape* Separate registration required; click here for details.

FRIDAY, AUGUST 17


DETECTION, CHARACTERIZATION AND CONTROL OF PROCESS- AND PRODUCT-RELATED IMPURITIES8:25 Chairperson’s Remarks



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Richard Cavicchi, PhD, Research Physicist, Biomolecular Measurement Division, Material Measurement Laboratory, National Institute of Standards and Technology

8:30 High-Throughput and Automated Impurities Detection Methods for Accelerated Biotherapeutic Manufacturing ProgramsDerek Ryan, PhD, Senior Scientist, Analytical Development, KBI Biopharma, Inc.With increasing demands to reduce the biotherapeutic time to clinic, process development efforts have adopted increasingly accelerated platforms. To keep up with this accelerated pace, a combination of high-throughput and automated methods have been developed to deliver insight into host cell protein and high molecular weight impurities clearance during process development.

9:00 Appearance of a Novel Basic Charge Variant during Redevelopment of a Monoclonal Antibody Production Process: The Path to Identification and ControlJeremy Pike, MS, Development Scientist, Early Stage Process Development, Alexion PharmaceuticalsRe-development of a monoclonal antibody production process resulted in a major change in a product-related charge variant. Multiple process development studies were executed to explore the potential source, assess potential impact to product safety and efficacy, and identify process levers for control. Multi-functional cooperation was crucial in identifying the potential cause and product impact, as well as the most likely modification responsible for this product quality shift.

9:30 Evaluation of the Simple Plex Ella Platform For Accelerating Process Development and Supporting Next-Generation ManufacturingCaitlin Kramer, MSc., Associate Scientist, Process Development Analytics, Bristol-Myers SquibbThe ProteinSimple Ella instrument was evaluated for its potential application in substituting for traditional ELISA impurities assays. In-process samples for multiple BMS mAbs were examined for dilutional linearity and spike recovery. Ella was evaluated from both a high throughput process development perspective and a process analytical technology perspective, for future use in next-generation manufacturing scenarios.


10:30 Monitoring Oxidative Damage in Therapeutic Proteins by Spectroscopic MethodsSambit Kar, PhD, Principal Scientist & Head, Biophysics Center of Excellence, Molecular & Analytical Development, Bristol-Myers Squibb Co.In this presentation, we will discuss oxidative stress on therapeutic proteins and share strategies for monitoring degradation pathways and identifying degradation products.

11:00 Development of High-Throughput Assays to Track Lipase-Induced PS80 Degradation in Support of Commercial Process DevelopmentJay West, PhD, Scientist II, Process Development Analytics, Bristol-Myers SquibbLipase-induced degradation of sorbate-based excipients/surfactants poses a critical risk for long term stability of therapeutic proteins and monoclonal antibodies. Our

discovery of rapid PS80 loss required timely analytic method development. Here we share two novel methods: LC/MS measurement of PS80 mono-oleate species, and a fluorescence method to measure total lipase activity. These were implemented in a high-throughput complimentary format to develop commercial processes for multiple therapeutic proteins.

11:30 Relating Measurements Made by Orthogonal Methods for Subvisible Particle CharacterizationRichard Cavicchi, PhD, Research Physicist, Biomolecular Measurement Division, Material Measurement Laboratory, National Institute of Standards and TechnologyOrthogonal measurements are often used with the goal of verifying an assessment of particle count and size distribution. While such methods usually agree when calibration beads are used, significant discrepancies have been noted when characterizing protein aggregates. To determine the causes of the differences, we present case study experiments that perform simultaneous orthogonal measurements on a per particle basis. We also estimate an important parameter, the spatially averaged protein aggregate density, which plays an important role in measurements such as the resonance mass technique. The results may lead to calibration parameters for relating measurements.



1:15 Session Break

DETECTION, CHARACTERIZATION AND CONTROL OF VIRUSES AND PATHOGENS1:25 Chairperson’s RemarksAnne Stokes, PhD, GSK Fellow and Director, TSE and Virus Control, GMP Operations, Biopharmaceutical Development and Supply, GlaxoSmithKline

1:30 FEATURED PRESENTATION: TSE and Virus Control Framework for the Development and Manufacture of BiopharmaceuticalsAnne Stokes, PhD, GSK Fellow and Director, TSE and Virus Control, GMP Operations, Biopharmaceutical Development and Supply, GlaxoSmithKlineBiopharmaceutical products are inherently at risk of contamination from adventitious or endogenous viruses, novel microbial agents and transmissible spongiform encephalopathies (TSE, or prion diseases), which can be introduced into manufacturing processes. These agents pose a risk to the manufacturing facility and patient safety. This presentation will outline the control framework to prevent these agents in the manufacturing process and will discuss the tools and strategies for detection, characterization and control.



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2:00 Second Generation Reference Materials for Adventitious Virus Detection by MetagenomicsEdward Mee, DPhil, Senior Scientist, Live Viral Vaccines, Virology, National Institute for Biological Standards and ControlMetagenomics offers great potential for improved adventitious virus screening; however, many practical challenges exist, including cost, complexity and the difficulty in defining limits of detection for unknown viruses. Well-characterized reference materials containing representative virus genome, capsid and envelope structures will enable meaningful inter-laboratory comparisons, support method development and act as process run controls.

2:30 Next-Generation Sequencing: A New Approach to Adventitious Virus DetectionCassandra Braxton, PhD, Senior Manager, Global QC Virology, BiogenCell culture-based methods are limited in their detection capabilities in that they are only able to detect viruses that cause cytopathic effect, hemadsorption, or hemagglutination. Biogen is exploring next-generation sequencing (NGS) as a new approach to adventitious virus detection. This presentation will provide an overview of NGS, considerations for implementation in a GMP environment, and evaluation against current adventitious virus detection methods.

3:00 Predicting MVM Clearance by Nanofiltration and Anion Exchange Chromatography Using a Non-Infectious “Mock Virus Particle”David Cetlin, Founder & CEO, MockV Solutions LLCViral clearance is an expensive and logistically challenging aspect of bioprocess development. A non-infectious “Mock Virus Particle”, engineered to physicochemically resemble MVM, was used as a spiking surrogate during small scale Virus Filtration and Anion Exchange Chromatography experiments. The results demonstrate the validity of utilizing this surrogate as a cost-effective way to predict or optimize for MVM clearance as part of process development and characterization studies.




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Analytical & Quality The week-long Analytical & Quality stream offers in-depth updates on critical steps in biopharmaceutical development that impact product quality, safety and regulatory compliance. Separate meetings will focus on the detection, analysis and removal of host cell proteins, early stage analytical development and the implementation of process characterization and control strategies. Over fifty in-depth presentations will give attendees insight into the best practices being applied across the industry.

AUGUST 13-14AGENDA Host Cell Proteins

AUGUST 15-16AGENDA Accelerating Analytical Development

AUGUST 16-17

AGENDA Process Characterization & Control

2018 ANALYTICAL & QUALITY TRACKS



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3RD ANNUAL

Host Cell ProteinsDetection, Analysis and Control


MONDAY, AUGUST 13


9:00 - 11:30 Recommended Short Course*SC5A: Introduction to Host Cell Proteins: Analysis, Characterization, Risks, and Requirements* Separate registration required; click here for details.


HCP REGULATIONS AND STANDARDS1:00 pm Chairperson’s Opening RemarksDenise Krawitz, PhD, Principal Consultant, CMC Paradigms LLC

1:10 KEYNOTE PRESENTATION: New USP Initiatives to Support Host Cell Protein Analytical MethodsMaura Kibby, PhD, Director, Global Biologics, U.S. PharmacopeiaImpurities such as host cell proteins (HCPs) must be cleared from recombinant biotherapeutics to minimize safety concerns and ensure the quality of the drug substance. HCP ELISA’s can miss specific proteins that may persist, and therefore the use of orthogonal methods has increased. USP’s HCP Expert Panel developed General Chapter <1132> to describe best practices for measurement of HCPs. This presentation will include an update on USP standards to support characterization and measurement of specific HCPs.

1:45 FEATURED PRESENTATION: Regulating Host Cell ProteinsErika Friedl, PhD, Quality Expert, Hematology and Transfusion Medicine, Paul-Ehrlich-InstituteSpecific HCP regulatory guidance is outlined in the European Pharmacopoeia and the US Pharmacopoeia, which provide an important regulatory framework. The interpretation of the regulatory framework and its flexibilities will be discussed. Case studies will be presented to outline the expectations regarding the HCP control strategy, the specification setting and the type of HCP assays used during product development and in the licensing process.

2:15 Use of Host Cell Protein Risk Assessments to Help Ensure Patient Safety, Product Quality, and Manufacturing Process ConsistencyDenise Krawitz, PhD, Principal Consultant, CMC Paradigms LLCWith improvements in analytical technologies, such as mass spectrometry, identification of individual HCP impurities has become more commonplace during clinical development over the past several years. As we learn more about HCP impurities, we can better understand the impact HCPs can have on the stability and safety of biotechnology products. This presentation will cover HCP risk assessment strategies to help ensure patient safety and product quality throughout product development.


HCP ASSAY DEVELOPMENT3:15 Same but Different: A Case Study of HCP Comparability for an In-Licensed ProjectFeny Gunawan, PhD, Scientist, Analytical Operations, GenentechThis presentation will focus on a case study in which we evaluated the HCP comparability of an in-licensed project. The challenges that we were facing were two-fold: 1) to determine the HCP comparability of the in the in-house produced material to the previous company’s material that was used in the pivotal trial, 2) to assess the suitability of HCP ELISA – a commercial HCP ELISA was compared to our platform HCP ELISA.

3:45 The Journey to Replace an Existing HCP Assay - Starting with the Right AntigenEmily Menesale, Senior Associate Scientist, Analytical Development, BiogenMeasuring residual HCP is essential for the bioproduction process. HCP assay results are reagent-dependent by nature, so there are significant challenges when it comes to replacing existing HCP reagents. Here, we describe the careful selections taken in our antigen processing and characterization to ensure an optimized antigen prep. This antigen ultimately led to antibody production and a successful HCP assay development that resulted in a suitable HCP replacement method.

Sponsored by4:15 Common Mistakes and Myths of HCP Analysis - Integration of Orthogonal Methods to Detect Individual Downstream HCPs Ken Hoffman, President, Cygnus TechnologiesVarious regulatory guidance articulate better practices in HCP analytics. However, the repertoire of HCP analytics still contains several outdated conventional practices. This talk identifies several mistakes and myths regarding HCP analytics, discusses proper methods to generate HCP antibodies and develop ELISAs. With Antibody Affinity Extraction (AAE) to replace 2D WB, and improvements in Mass Spectrometry, the identification of individual downstream HCPs is now within our capability and should be the goal of any well-characterized biologic.



7:00 End of Day



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3RD ANNUAL



TUESDAY, AUGUST 14


IMPROVING HCP ANALYSIS WITH MASS SPEC7:55 Chairperson’s RemarksYan-Hui Liu, PhD, Merck & Co., Inc.

8:00 Making the Most of Mass Spectrometry in Host Cell Protein Detection and ControlChristopher Yu, PhD, Principal Scientist, Genentech, A Member of the Roche GroupIdentification of specific residual host cell proteins (HCP) in biopharmaceutical products by mass spectrometry addresses potential gaps in immunodetection coverage. This study examines the technical aspects of HCP quantitation by mass spectrometry, and demonstrates feasibility of fast and relevant quantitation for optimal process development. We also discuss the importance and potential opportunities in defining the quantitative ranges of HCP for safe and efficacious use of biopharmaceuticals.

8:30 Qualification of an Absolute Quantification Workflow for HCP Determination by LCMSVeronika Reisinger, PhD, Lab Head, Biologics Technical Development and Manufacturing, NovartisELISA is the standard method for HCP detection but is usually not capable to identify and quantify single HCPs. For tracking of a specific HCP, an absolute quantification workflow based on LCMS/MS was developed and qualified regarding different parameters like linearity or repeatability. The method was applied to support process optimization as well as increase knowledge on the final drug substance.

9:00 Determination of Proteins Detected by Host Cell Protein ELISA Assays Using Mass SpectrometryMichelle Busch, PhD, Scientist, Bioanalytics Characterization, SanofiMass spectrometry is typically used as an orthogonal HCP characterization technique while an ELISA method is used as the release assay. Sometimes, ELISA assays have been shown to be insensitive to relatively abundant HCPs. In this study, the antigens used to raise ELISA antibodies were analyzed to measure amounts of HCPs and then the antibodies were used to purify HCPs from products to better understand ELISA assay results.

9:30 Sponsored Presentation (Opportunity Available)


OPTIMIZING HCP ANALYSIS10:30 Tracking Host Cell Protein Impurities in Biopharmaceuticals Using Mass SpectrometryRegina Kufer, PhD, Senior Associate Development Analytics, Roche Diagnostics GmbHRecently a MS-based platform was developed for host cell protein (HCP)

characterization to support purification process development. This technique provides an orthogonal and complementary approach to traditional HCP analysis by enzyme-linked immunosorbent assays (ELISA). In contrast to the ELISA, LC-MS/MS analysis provides the identity of HCPs. Here, we present a simple and powerful strategy combining our platform LC-MS/MS with a proteomic approach to increase sensitivity and number of detected HCPs.

11:00 Advances in HCP Analysis for the Quality Control LaboratoryJamie Rusconi, PhD., Senior Analytical Scientist, Analytical Sciences, Regeneron Pharmaceuticals IncWhile ELISA assays generally offer the advantages of broad HCP recognition and sensitivity, they also suffer from a limited linear dynamic range and multiple steps requiring analyst “hands on” time. The ELLA instrument from ProteinSimple has been developed as an alternative to the traditional immunoassay format. In this presentation, we will explore the application of the ELLA instrument from ProteinSimple as an alternate approach to ELISA for HCP analysis.

11:30 High Throughput LC-MS Platform for HCP Characterization in Support of Process DevelopmentSuli Liu, PhD, Scientist II, BiogenAs an orthogonal method to ELISA, LC-MS based analytical approach offers unique features of identification and quantitation of individual HCPs. A high throughput, high sensitivity and robust LC-MS based platform has been developed to support HCP clearance monitoring during process development. This platform relies on 1D LC coupled with advanced mass spectrometry and combined with ‘Hi 3’ quantitation method achieves detection and quantitation of HCPs in a single run. The method has been optimized for up to 19 samples per day with low ppm detection limit. This presentation will discuss the method development, qualification and a case study.

Sponsored by12:00 pm Characterization, Optimization and Application of an LC-MS Based Workflow for HCP Identification and ControlMichael Schirm, PhD, Associate Director, Research & Development Proteomics, Caprion Biosciences, Inc.Mass spectrometry (MS) enables identification and quantitation of total and individual HCP in biotherapeutic products, and represents an orthogonal method to ELISA. Examples will be presented showing use of semi-quantitative identification of individual/total HCP (LC-MS/MS) and absolute quantitation of HCP (LC-MRM/MS), as applied to monitoring of process changes/improvements, scale-up, batch uniformity, clearance, and comparison of Biosimilars vs. Innovators. Caprion’s HCP platform features customizable organism/process-specific databases, highly controlled analytical processes and reproducible robust detection (to ~1ppm).

Sponsored by12:30 Luncheon Presentation: Host Cell Proteins-Integrated Analytical Solutions Using Immunoassays and Mass SpectrometryAndrew Hanneman, PhD, Associate Director, Biologics Testing Solutions, Charles River Laboratories



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3RD ANNUAL




HOW HCP ANALYSIS IMPACTS BIOPROCESSING1:55 Chairperson’s RemarksYan-Hui Liu, PhD, Merck & Co., Inc.

2:00 Evaluation of Analytical Technology to Accelerate Process Development and Support Next-Generation ManufacturingCaitlin Kramer, PhD, Associate Scientist, Process Development Analytics, Bristol-Myers SquibbInstruments were evaluated for their potential application in substituting for traditional ELISA impurities assays. The options were evaluated from both a high-throughput process development perspective and a process analytical technology perspective, for future use in next-generation manufacturing scenarios.

2:30 Identification and Impact of Individual Host-Cell Proteins in CHO-Based BiomanufacturingAbraham M. Lenhoff, Allan P. Colburn Professor, Department of Chemical and Biomolecular Engineering, University of DelawareOverall levels of host-cell proteins (HCPs) in biomanufacturing are routinely monitored by methods such as ELISA. However, even when such levels are reduced to acceptable levels, individual HCPs may be problematic within a bioprocess or may impact drug product quality. This presentation will discuss the identification of such problematic HCPs, possible reasons for their persistence, and remedial actions that may be employed to address the deleterious effects.

Sponsored by3:00 An Improved CHO HCP Detection System for BiopharmaceuticalsMindy Wan, PhD, Technical Director, Business Development, KBI Biopharma, Inc.KBI has applied a unique strategy to develop an improved CHO HCP detection system for a variety of protein therapeutics expressed in CHO cell lines widely used in biopharmaceutical industry. KBI’s CHOP ELISA method exhibits superior dilution linearity and matrix flexibility for detecting CHOPs present in purification process intermediates and final products. The reagent used in this ELISA shows >70% coverage for a pooled null harvest generated from several CHO cell lineages.


4:15 Linking HCPs to Bioprocess PerformanceYing Zhang, PhD, Senior Scientist, Analytical Research & Development, PfizerLC-MS/MS has emerged as an orthogonal approach to ELISA for analyzing HCPs, providing both qualitative and quantitative information on individual HCPs. This presentation will discuss the use of LC-MS/MS to improve the understanding of bioprocessing.

4:45 Downstream Process Optimization Strategies to Remove Persistent HCP ImpuritiesNicholas Levy, PhD, Investigator, GSKHigh levels of fragment were observed during early phase process development and clinical manufacturing of an IgG1 monoclonal antibody (mAb). Fragment concentration increased across the downstream process, and stability testing demonstrated rapid fragmentation of the mAb in bulk drug substance at both 5 and 25 ºC.




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5TH ANNUAL

Accelerating Analytical DevelopmentOptimizing the Speed and Efficiency of Key Analytical Steps in Biotherapeutic Development




OPTIMIZING THE SPEED AND EFFICIENCY OF ANALYTICAL DEVELOPMENT8:05 Chairperson’s RemarksChristina Vessely, PhD, Senior Consultant, Analytical and Formulation Development, Biologics Consulting

8:15 KEYNOTE PRESENTATION: Balancing Speed and Resource Considerations in the Selection of Fit-for-Purpose Assays at Each Stage of DevelopmentJennifer F. Nemeth, PhD, SCPM, Director, Biophysics, Structural Characterization, Biologics Discovery Sciences, Janssen Research & DevelopmentThe need for fit-for-purpose biophysical assays at each stage of development is well established. Early discovery requires fast assessments using limited material. In contrast, later development studies are routine, validated, and robust with minimal material constraints. Depending on the function, studies operate in a regulated environment requiring GLP or GMP conditions. Here, the assay path for large molecule analyses are examined, with an emphasis on scale, desired information, and impact to development.

9:00 Best Practices for Comparability Analysis Following Method Changes and Method BridgingChristina Vessely, PhD, Senior Consultant, Analytical and Formulation Development, Biologics ConsultingAnalytical methods and specifications evolve across the product development lifecycle, including the addition of new analytical methods or improvements to current methods. As the need arises for process changes to support ongoing clinical studies, it is necessary to demonstrate that materials produced from the post-change process are comparable to those produced in the pre-change process. This presentation discusses strategies for bridging across method changes to establish product comparability.

9:30 Feasibility and Challenges in Machine Learning Approaches to Predicting Biophysical Properties from Sequence and StructureTileli Amimeur, Data Scientist, Molecular Design, Just BiotherapeuticsBiotherapeutic development is a slow and costly process. We can make use of data to improve screening, engineering, and optimization of molecules for downstream properties. Predictive methods can reduce the time and cost of development while delivering higher quality biologics. We explore the feasibility and challenges in building machine learning models for predicting biophysical properties of therapeutic antibodies from sequence and structure.


10:45 Regulatory Considerations in Determining Analytical Strategy for Phase I ProgramsVaneet K. Sharma, PhD, Manager, Analytical Development, Vaccine Development & Manufacturing, International AIDS Vaccine Initiative (IAVI)This presentation will outline key regulatory considerations in determining analytical strategy especially for exploratory and Phase I programs. Analytical strategies for the assay development, assay qualification and characterizing the critical quality attributes (CQAs) relevant to Phase I/II clinical trial material will be discussed. A case study will be presented to demonstrate the application of the regulatory accepted phase-appropriate analytical characterization to support HIV vaccine development.

11:15 An Efficient Analytical Development Strategy Enabling Expedited Development of Therapeutic ProductsXiaoyang Zheng, PhD, Principal Scientist, Biopharmaceutics Development, Sanofi USAn efficient approach for a new analytical method development consists of five stages with clear deliverables: method definition, platform selection, development and optimization, evaluation, and documentations. We will present case studies to show how to streamline method development using previous knowledge, job aids, platform, and templates to define performance requirements, identify key variables, increase method robustness, design-appropriate control strategy, and reduce development time/costs.

Sponsored by11:45 How to Use Analytical Tools for Process Development Xinfang Li, PhD, Vice President, Process Development, MabPlexProcess development plays a key role in drug development. Robust process defines product quality and manufacturing consistency. A case study is presented here to demonstrate how the process parameters are defined based on analytical results.


1:00 Session Break

EMERGING METHODS AND INSTRUMENTS TO SUPPORT ACCELERATED DEVELOPMENT1:45 Chairperson’s RemarksLintao Wang, PhD, Principal Scientist and Mass Spec Group Leader, Analytical and Pharmaceutical Science, ImmunoGen, Inc.

1:50 Capillary Electrophoresis Sodium Hexadecyl Sulfate (CE-SHS): A Novel Approach to Characterizing the Purity of a Therapeutic ProteinJeff Beckman, PhD, Senior Scientist, Bristol-Myers SquibbAccelerated commercial process development is the new norm in pursuit of meeting patient requirements, and analytical data integrity is key to making speed to market possible. We present data from a newly developed high-resolution CE-SHS technology that markedly improved data integrity relative to standard approaches (e.g. CE-SDS). This CE-SHS-based technique has become integral at BMS for relevant accelerated assets.



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5TH ANNUAL



2:20 Novel IR Microfluidic Device for Secondary Structure Assessment of an Antibody and its ADCKaran Shah, Principal Development Associate, Analytical and Pharmaceutical Sciences, ImmunoGen, Inc.Protein secondary structure is an important aspect of higher order structure characterization of biotherapeutics and can provide vital information about stability, aggregation, comparability, etc. It becomes even more critical in ADCs since folding patterns of parent antibody may be altered owing to conjugation of hydrophobic payload and can possibly alter biological properties. This talk will cover analysis of antibodies and corresponding ADCs using a new Microfluidic Modulation Spectrometry (MMS) technique and comparison with alternative approaches.

2:50 Online Peptide Immuno-Affinity Enrichment (PIE) LC-MS Application in Biodistribution Studies of Protein TherapeuticsJessy Fan, PhD, Scientist, AmgenFor protein therapeutics, quantitative analysis of tissue distribution facilitates the understanding of in vivo exposure to response relationship. Online Peptide Immuno-Affinity Enrichment (PIE) LC-MS workflow has been developed to complement currently available technologies for biodistribution of protein therapeutics. The unique PIE feature of multiplexing capabilities and translatability from preclinical to clinical studies will be presented here, providing additional evaluation parameters of selecting protein therapeutics and their development.

3:20 New Developments in Reducing Sample Size Requirements for Common AssaysJoshua Laber, PhD, Postdoctoral Fellow, AbbVieCurrent techniques are unsuitable for high-concentration viscosity measurements in early stage candidate molecule screening due to sample limitations. Here we introduce the iBEACON (integrated Buffer Exchange And CONcentration) which scales traditional tangential flow filtration (TFF) to <2 microliter final volume with 200-fold protein concentrations and provides continuous viscosity, shear, and filtrate flux rate measurements. This fully automated system also performs unattended in situ buffer exchanges for formulation screening of multiple samples.




7:30 End of Day

THURSDAY, AUGUST 16


SOFTWARE AND DATA MANAGEMENT CHALLENGES8:25 Chairperson’s RemarksAlejandro Carpy, PhD, Laboratory Head Biochemistry, Roche Innovation Center Munich, Germany

8:30 Electronic Lab Environment: Software and Data ChallengesRicha Sarin, Scientist, Technical Development, BiogenElectronic lab environments can entail a multitude of heterogeneous systems to accommodate diverse scientific applications, resulting in significant challenges in interoperability. Two of those challenges include easily extracting knowledge and value from the data and limited exchange and visibility into the data. This presentation will focus on use cases aimed at addressing such challenges within an analytical development laboratory; automating delivery of data from our external partners and utilizing data analytics to enable swift and informed decisions.

9:00 Accelerating Insights through Aggregation of Formulation Data and Metadata from an Integrated Process Development Electronic DatabaseShabnam Molloy, Senior Associate Scientist, AmgenThe rise of electronic laboratory notebook and cloud data storage systems have enabled companies to structure the way data are collected to facilitate searching using key parameters. Visualization software tools can be configured to integrate cloud-based searches to make data collection and analysis easier for the average scientist without the need for additional manipulation. In this case study, we present an example of how this can be achieved to make faster, informed program decisions.

9:30 The Use of an Automation Platform to Reduce Assay Transfer Burden and Timeline - An Impurity Assay Development StudyWilliam Grimm, Scientist, Process Development Analytics, Bristol-Myers SquibbThe process of analytical transfer from Development labs and QC labs to multiple testing sites is a critical activity during all phases of drug development and commercial assets. Automation plays a major role in simplifying and expediting many plate based assays during Development. This work shows how an ELISA based assay with an automated liquid handler can be optimized to align with the manual best practice of a QC lab.




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5TH ANNUAL



ANALYTICAL DEVELOPMENT CHALLENGES OF NEW MODALITIES10:45 Correlation of Analytical Methods for Next-Generation Biologics from Molecular Assessment to Clinical DevelopmentAlejandro Carpy, PhD, Laboratory Head Biochemistry, Roche Innovation Center Munich, GermanyIn order to accelerate and optimize method development, we performed an in-depth assessment of the analytical data generated from lead candidate selection to technical development using customize methods and a combination of platforms developed for complex formats. Furthermore, we analyzed data collected for different molecular formats to ensure correct product quality and pCQA assessment across platforms during the analytical method development.

11:15 Analytical Development and Challenges for Bispecific AntibodiesXiaoyu Yang, PhD, Principal Scientist, MerckBiologic drugs have evolved into an ever-growing diversity of modalities, such as monoclonal antibodies, bispecific antibodies, and peptides/proteins. Bispecific antibodies have received wide attention as a promising immunotherapeutic agent. Analysis of bispecific antibodies presents challenges because multiple antibody forms are potentially generated in cell culture. The analytical strategy and some example methods implemented to screen cell culture clones, monitor purification steps, and evaluate antibody purity will be presented.

11:45 Analytical Platforms in an Era of Novelty and New ModalitiesDaniel McDonald, PhD, Researcher, GenentechThe increasing complexity of therapeutic protein modalities, combined with shrinking development timelines has put a strain on the platform method concept. This presentation discusses a nimble approach of coupling automated development software with a malleable platform method. With this approach, previously validated parameters can be optimized to create a product specific method. Ultimately, this will allow analytical platforms and their benefits (time, resource reduction) to be used for diverse modalities






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4TH ANNUAL

Process Characterization and ControlA Best Practices Forum for the Translation of Process Understanding into Control Strategiesfor Maintaining Quality throughout the Product Lifecycle


THURSDAY, AUGUST 16




1:55 Chairperson’s RemarksMin Zhang, PhD, Director, Manufacturing Sciences and Technology, AstraZeneca

2:00 KEYNOTE PRESENTATION: ‘Real-Time’ Monitoring of the Structure of a Monoclonal Antibody during Chromatographic Elution from a Protein A Affinity ColumnTim Dafforn, PhD, Professor, Biotechnology, University of Birmingham, United KingdomThe biopharma industry’s increased familiarization with continuous processing technologies has led to the emergence of facilities operating semi-continuous or continuous production trains. However, for the benefits of continuous processing to be realised, it will be necessary to develop novel process analytical technologies (PAT). In this presentation, we describe the development of an automated high-throughput PAT system combining circular dichroism and fluorescence spectroscopies.

PROCESS CHARACTERIZATION2:45 The Challenges of Deploying a QBD Approach for Accelerated and Breakthrough Status ProgramsNaveen Pathak, Director, Process Development, ShireDeploying QbD for breakthrough and accelerated products presents both a timeline challenge and an enabling opportunity. Product and process understanding, the foundation of Qbd strategy, is time and resource intensive. Strategies will be discussed that allow for acquisition of products and process understanding in a step wise fashion while acceleration of PPQ initiation. The enabling factors for such a strategy and the value proposition will be presented to demonstrate that QbD approach is the optimal approach to secure accelerated approval.

3:15 Cell Culture Media Characterization Strategy and Related Challenges in Commercial ManufacturingMin Zhang, PhD, Director, Manufacturing Sciences and Technology, AstraZenecaIn overall upstream process characterization strategy, a well-defined cell culture media characterization plan is deemed crucial and highly desirable to ensure a robust commercial manufacturing process. The media characterization strategy generally considers raw material variability (lot-to-lot, vendor-to-vendor), the optimal media preparation process, acceptable ranges for key scaling parameters, and related media preparation operational variability. This presentation outlines the media characterization strategy at AZ and how it impacts commercial manufacturing.



4:15 Mass Spectrometric Monitoring of Critical Quality Attributes to Support ADC Process CharacterizationLintao Wang, PhD, Associate Director, Analytical and Pharmaceutical Development, ImmunoGen, Inc.Biotherapeutic antibody-drug conjugates (ADCs) are tumor-targeting monoclonal antibodies covalently linked with potent cytotoxic agents. Due to the molecular complexity of ADCs, it is critical to understand, characterize and qualify the conjugation process to deliver quality products. For this purpose, mass spectrometric methods were developed and applied in process characterization studies to gain full understanding of the effects of the process parameters on the quality attributes of an ADC.

4:45 Targeted Profiling: Using NMR beyond FingerprintingMarc Aucoin, PhD, Associate Professor, Chemical Engineering, University of Waterloo, CanadaTogether with a well curated database, an NMR spectra can be deconvoluted to reveal the composition of complex solutions such as cell culture media. In this talk, we will discuss the benefits and pitfalls of this technology in the characterization of insect and CHO cell culture processes. For the former, we have examined the contributions of yeast extract to chemically defined incest cell media, while for the latter, we have tracked compounds such GlutaMAX™ and acetate to better understand nucleotide-sugar precursor feeding for more uniform glycosylation of antibodies.

5:15 End of Day

6:00 - 9:00 Recommended Dinner Short Course*SC13C: Implementing Phase Appropriate GMP for Manufacturing Biologics and Cell Therapy Products* Separate registration required; click here for details.

FRIDAY, AUGUST 17


CRITICAL QUALITY ATTRIBUTES AND SPECIFICATION SETTING8:25 Chairperson’s RemarksUdayanath Aich, PhD, Principal Scientist, Sanofi Genzyme



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4TH ANNUAL



8:30 Data-Driven Determination of Potency Critical Quality Attributes for an ADCTaro Fujimori, PhD, MBA, Associate Director, Protein Analytics, Science and Technology Biologics, AbbVie Bioresearch CenterADC-123 is an antibody drug conjugate comprised of an antibody (mAb-123: targets Receptor XYZ expressed on tumor cells) conjugated to a cytotoxin. ADC-123 has three activities that might contribute to anti-tumor activity: Delivery of cytotoxin to tumor cells with elevated levels of Receptor XYZ, Inhibition of Receptor XYZ signaling and Fc effector function. The primary mechanism of action for ADC-123 is delivery of the cytotoxin to tumor cells with elevated levels of Receptor XYZ.

9:00 Best Practices for Specification Setting: Regulatory Expectations for SpecificationsDarrin Cowley, PhD, Head, Development Quality, Biologics, AstraZenecaSpecification setting remains a scientific challenge for the biotechnology industry as there is a balance between product-specific knowledge, which can be limited due the numbers of batches produced and/or accelerated programs versus prior knowledge from similar molecules. Understanding of ‘required’ tests (e.g. appearance, microbial, content, etc.) and critical quality, consistency and stability attributes enables a lot release strategy. The functionality of any device is important for combination products.

9:30 Microchip Electrophoresis-MS for Rapid Product Quality Determination and ControlSeth Madren, PhD, Scientist, BiogenTherapeutic mAbs are highly heterogeneous, and Critical Quality Attributes (CQAs) of monoclonal antibodies need to be monitored or characterized to assure product safety and efficacy. In this talk, we present a high-throughput CE-MS method that enables not only near-real time CQA monitoring during cell culture process, but also fault detection as a result of wrong feed or contaminant. Potentially reducing failed batches and permitting possible corrective actions.


CHALLENGES IN PROCESS CHARACTERIZATION AND CONTROL10:30 Real Time Online Chromatography Monitoring of Product Quality Attributes for Biologics Continuous Manufacturing ProcessGang Xue, PhD, Scientific Director, AmgenThe ever-diversifying therapeutic modalities drive for modular and flexible bio-manufacturing. Process Analytics evolve as critical enabling element of the CM process. For the first time, we leverage a multi-functional automation system to directly take samples from the different stage of bio-process, purify, denature, derivatize and digest the samples before injecting onto the UHPLC and UHPLC/MS systems, one for online intact protein analysis, the other for Multiple Attribute Method (MAM) analysis for critical PTM PQAs.

11:00 Roadmap of In-Line Monitoring and Real Time Release (ILM-RTR): A Collaboration Effort by Biomanufacturers, Suppliers and Regional Research HubsUdayanath Aich, PhD, Principal Scientist, Sanofi GenzymeThe presentation describes a roadmap strategy published by BPOG in July 2017 with active collaboration of Biopharma industry and supply partners. The roadmap implementation is focused on the development of a user requirement strategy (URS) to prioritize the most relevant critical quality attributes (CQA) and critical process parameters (CPP) focusing on the critical control points and future short-, mid- and long-term goals to develop and apply innovative technologies for real time release.

11:30 Late Stage Cell Culture Concept – From Risk Assessments to Process CharacterizationEugen Probst, Senior Scientist, Late Stage Upstream Development, Boehringer Ingelheim, Germany For the commercialization of the upstream process a comprehensive late stage development concept is required. The process knowledge gained during development and clinical manufacturing runs is compiled and utilized in a series of risk assessments to define the process characterization studies. The presentation summarizes the requirements and challenges for knowledge-based risk assessments, establishment of scale-down models and extensive process characterization.

Sponsored by12:00 pm Profiling and Monitoring of Quality Attributes in Biopharmaceutical Characterization, Process Development and QC Weibin Chen, Director, Scientific Operations, Waters CorporationAiming to support QbD, gaining greater understanding of biotherapeutics, an increasing trend of moving MS into lab settings traditionally associated with optical-based assays has been observed. This has facilitated the introduction of LCMS-based Multi Attribute Monitoring methods to increase productivity. Challenges associated with these approaches include monitoring PQAs in large datasets, setting specifications, and transferring methods from development to QC. Herein we will demonstrate how these challenges can be addressed with a compliance-ready platform solution.


1:15 Session Break

CONTROL STRATEGIES1:25 Chairperson’s RemarksParag Kolhe, PhD, Group Leader and Associate Research Fellow, Pfizer



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4TH ANNUAL



1:30 A Systematic Approach to Establish Drug Product Control Strategy during Process CharacterizationParag Kolhe, PhD, Group Leader and Associate Research Fellow, PfizerThe International Conference on Harmonization (ICH) Q8 (R2), Q9, Q10 and Q11 guidelines provide complementary guidance on pharmaceutical development, quality risk management, and quality systems, respectively, to ensure product quality using a scientific and risk-based approach. Process and product understanding are building blocks of control strategy to ensure consistent product quality. A case study will be presented which outlines the systematic approach that was taken for development of a vaccine.

2:00 Continuous, Online Antibody Quality Monitoring Using a Nanofluidic Device for Continuous BiomanufacturingTaehong Kwon, Graduate Research Assistant, Electrical Engineering and Computer Science, MITContinuous biomanufacturing is a growing trend in biopharmaceutical industry. Monitoring the critical quality attributes of biologics is essential at all stages of manufacturing process. We recently developed a nanofluidic system for continuous-flow multivariate protein analysis for real-time critical quality assessments. In this talk, we introduce our latest work on the integration of perfusion culture using a membraneless microfluidic cell retention device with nanofluidic system enabling real-time product quality assessments.

2:30 Upstream Process Control Strategy: Beyond Keeping Consistency with the PastJuhong Liu, PhD, Independent CMC ConsultantAdvances in analytics and understanding of clinically relevant product quality attributes indicate current control strategy may no longer be sufficient to identify drifts before products reach patients. Recent events demonstrated unintentional or seemingly innocent changes to upstream processes can impact these attributes. Updates to existing process and product controls to rapidly discover product quality drifts not covered in the initial approval may be necessary.

3:00 A Systematic Approach to Establish Upstream Control Strategy During Process CharacterizationDaniel Tong, PhD, Research Scientist, Gilead SciencesThis case study describes a systematic approach from risk assessment to the establishment of upstream control strategy for process characterization of a mAb production process characterization. Upstream process characterization studies were planned with DOE tool and executed in qualified SDM bioreactors. CPPs and KPPs were identified using both statistical analysis and practical significance analysis. NORs and PARs were defined for each CPP and KPP based on Monte Carlo simulation.




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Formulation & Stability The week-long Formulation & Stability stream brings together experts in formulation and analytical sciences and process scientists to share practical insights and case studies on rapid methods for analytical screening and development, biotherapeutic formulation development of traditional and novel formats, development of high-concentration protein formulations, screening tools and strategies to manage contaminants and impurities. The four conferences together will feature cutting-edge approaches for understanding and managing biologics product development, aggregations, excipient issues, high viscosity, prediction and screening of stability and quality issues, and impurities such as pathogens, host cell proteins, lipases and enzymatic degradation, aggregates, degradants and more.

AUGUST 13-14

AGENDA Rapid Methods to Assess Quality & Stability of BiologicsAGENDA Overcoming Formulation Challenges for Biopharmaceutical Development

AUGUST 15-16

AGENDA High-Concentration Protein Formulations

AUGUST 16-17

AGENDA Detection, Characterization and Control of Impurities in BiologicsAGENDA Training Seminar: Introduction to Biologics Formulation Development

2018 FORMULATION & STABILITY TRACKS



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6TH ANNUAL

Rapid Methods to Assess Quality & Stability of Biologics:Improving Prediction and Screening


MONDAY, AUGUST 13


9:00 - 11:30 Recommended Short Course*SC3A: Application of New Analytical Tools in Protein Formulation Development and Subvisible Particle Characterization* Separate registration required; click here for details.


FORMULATION AND ANAYTICAL DEVELOPMENT OF NEW MODALITIES1:00 pm Chairperson’s Opening RemarksNicholas Warne, PhD, Senior Director, Pharmaceutical R&D, BioTherapeutics Pharmaceutical Sciences, Pfizer, Inc.

KEYNOTE PRESENTATIONS1:10 Delivering Flexible Dosage Forms for Innovative Modalities and an Evolving Development PlanNicholas Warne, PhD, Senior Director, Pharmaceutical R&D, BioTherapeutics Pharmaceutical Sciences, Pfizer, Inc.The development of biopharmaceutical processes and products is predicated on assumptions of dose range, time to customize the dosage form and market preferences. While seeking efficient approaches to screening compounds to assess developability, the increased complexity of biologics requires innovative approaches to formulation and dosage form development. The balance of platform processes to create capacity to provide innovative formulation solutions to novel modalities will be the focus of this presentation.

1:45 Challenges in Downstream Processing and Analytics of a Large Diversity of Molecules/Formats in the Discovery SettingJohn E. Harlan, PhD, Principle Research Scientist, Global Protein Sciences, AbbVieDownstream processing in support of Biologics Drug Discovery provides challenges that are distinct from those encountered in Development. Here we will discuss the approach we have taken to develop platform processes that can address the large diversity of molecules we encounter in the Discovery setting in the shortened timelines needed to facilitate the Discovery process, including the fit for purpose analytical approach we take, and automated processes we have developed.

2:15 A Holistic Approach to Formulation Development: From Cell Line to Drug Product DevelopmentSonal Saluja, PhD, Scientist II, Technical Development, BiogenAn end-to-end approach is needed for formulation development. Process impurities

and heterogenous drug substance generated from upstream/downstream processes can negatively impact drug stability. This might not always be resolved by optimizing the formulation. Hence, a holistic development approach involving balancing yield, productivity, quality attributes and drug product stability is needed to guarantee success. This talk will highlight this concept using case studies.


CHARACTERIZATION OF ANALYTICAL PROPERTIES OF NOVEL FORMULATIONS3:15 Biophysical Characterization of Lipid Nanoparticles for RNA DeliveryMark Brader, PhD, Research Fellow, Drug Product Analytical Development, Moderna Therapeutics, Inc.Unlocking the potential of RNA therapeutics requires a sophisticated delivery system. Lipid nanoparticles can protect the RNA cargo from degradation in the bloodstream and facilitate cellular uptake. However, their physical and structural complexities present a special set of challenges for pharmaceutical analysis and control. This presentation will provide an overview of applicable biophysical approaches with an emphasis on colloidal properties and product development considerations.

3:45 Rapid Amino Acid Analysis (AAA) Methods for Protein Concentration in Novel Drug Delivery PlatformsScott H. Chamberlain, Senior Research Associate, Protein Analytical Chemistry, GenentechProtein concentration measurements by UV spectroscopy and other dye assay methods may be ineffective when biotherapeutics are conjugated to hydrogels, scaffolds or encapsulated in drug delivery devices. AAA can provide accurate quantification following assessment of the drug delivery platform prior to hydrolysis and analysis. Accelerated AAA analysis methods can allow for high-throughput and rapid determination of protein concentration. Several case studies will be discussed highlighting accelerated AA analysis.

Sponsored by4:15 High-Throughput Critical Quality Attribute Testing for Enhancing Speed to Market & Minimizing RiskBelinda Pastrana, PhD, CEO, Protein Dynamic SolutionsA walkaway, high throughput, array based platform will be presented that provides simultaneous assessment of eight CQA parameters, sample visualization and mechanistic determination of changes in therapeutic proteins. Up to 200 samples/day are assessed for developability and comparability across a range of formulations and stressors for improved pipeline success.




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7:00 End of Day

TUESDAY, AUGUST 14


RAPID SCREENING PLATFORMS FOR NOVEL AND TRADITIONAL BIOLOGICS7:55 Chairperson’s RemarksJay(Zhi) Guo, PhD, Senior Scientist, Biologics, AbbVie

8:00 Analytical Strategies for Development of Biopharmaceuticals: One Size Fits AllZahra Shahrokh, PhD, CMC Consultant and Chief Development Officer, STC BiologicsThe principles for development of biopharmaceuticals apply to different classes of molecules, yet each molecule is guaranteed to pose its own unique challenges. This talk focuses on best practices in pharmaceutical development, with a focus on analytical tools that could minimize future surprises. Case examples of excipient degradation and potential mitigation strategies will be discussed.

8:30 Developing a Screening Platform for Novel BiotherapeuticsJay(Zhi) Guo, PhD, Senior Scientist, Biologics, AbbVieNovel bi-functional biologics with a variety of formats to choose from (scFv, Fab, mAb, etc.) and fused to different payloads (cytokines, growth factors, a different scFv, etc.) is of immense interest for targeted delivery and improved efficacy. Payloads can be fused at different positions and spacer lengths and it is now apparent that payloads dominate production and stability of biologics, thus confounding drug-like properties (DLPs). To better understand structure-function relationship and rapidly advance candidates with good DLPs, we will discuss our throughput screening process.

9:00 Accelerating Potency Assays to Support Early Stage Product Quality and Formulation DecisionsNathan P. Oien, PhD, Senior Scientist, Analytical Development Department, KBI Biopharma, Inc.Potency assays are often unused during early stage biopharmaceutical development for various reasons including assay development timelines, high variability, and assay execution timelines. However, potency assays using a fully optimized assay could provide powerful data to help guide product quality and formulation decisions. Technologies and processes that exist to help increase throughput and decrease variability will be highlighted.

9:30 SELECTED POSTER PRESENTATION: Freeze-Drying of Highly Concentrated Proteins: Effect of Formulation and Processing on Phase Composition, Porous Structure and Reconstitution TimeShreya Kulkarni, PhD Candidate, Pharmaceutical Sciences, University of Connecticut


10:30 Chemical Stability Screening in Early Stage Antibody DiscoveryTingwan Sun, PhD, Senior Scientist, Protein Analytics, Adimab LLCIsomerization and deamidation of therapeutic leads can often delay development timelines and provide challenge and risks for downstream process. Sequence-based prediction to scan for NG and DG can often lead to false positive results. Here we report the chemical liability analysis of ~140 clinical stage antibodies, under forced degradation conditions.

11:00 Towards an Improved Predictor for Shelf Stability: Using Denaturants to Measure Interactions between Partially Folded ProteinsRobin Curtis, PhD, Senior Lecturer, Chemical Engineering and Analytical Science, University of ManchesterA key obstacle to predicting aggregation is difficulties in isolating the partially folded states believed to be key intermediates in the aggregation pathways. We have measured protein-protein interactions under chemically denaturing conditions as a surrogate parameter to reflect the colloidal stability for partially folded proteins under native conditions. We show the measurements provide an indicator for aggregation propensity and provide a complementary technique for assessing storage stability.

11:30 PANEL DISCUSSION: Assessing the Comparability and Stability of New Biologics and Novel Formulations• Examples for cell and gene therapy products• Guidance and expectations• Tools and techniquesModerator:Rajiv Nayar, PhD, President, HTD Biosystems, Inc.Panelists:Jay(Zhi) Guo, PhD, Senior Scientist, Biologics, AbbVieJohn E. Harlan, PhD, Principle Research Scientist, Global Protein Sciences, AbbVieMark Brader, PhD, Research Fellow, Drug Product Analytical Development, Moderna Therapeutics, Inc.Peter Fung, PhD, Senior Manager, Product Marketing, NanoTemper TechnologiesScott H. Chamberlain, Senior Research Associate, Protein Analytical Chemistry, GenentechZahra Shahrokh, PhD, CSO, STC Biologics

Sponsored by12:00 pm Dig Deeper into Protein Quality and Stability Lisa Adamiak, PhD, Applications Scientist, Marketing, Unchained LabsUnderlying all biologics development is finding the best protein candidates along with optimal formulation conditions to minimize aggregation while preserving stability.



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Whether you’re trying to understand more about your molecules earlier or later in your process, Unchained Labs can simplify your work. We’ll discuss how you can use a combination of applications like thermal melting and aggregation, sizing and polydispersity, and chemical denaturation to thoroughly characterize more biologics with less sample and faster than before.

Sponsored by12:30 Luncheon Presentation: A Quick Protein Quality Check that Will Improve Biotherapeutic Candidate Purification and Characterization WorkflowsPeter Fung, PhD, Senior Manager, Product Marketing, NanoTemper TechnologiesStarting with material of questionable quality for protein purification and characterization leads to irreproducible or ambiguous results. Transitioning between upstream and downstream workflows can be a challenge for bioprocessing researchers, particularly when the quality of the sample material is not known. We present a new platform that swiftly identifies sample quality and relative functionality in minutes complementing and guiding bioprocessing workflows—making go/no go decisions easy and quick—saving time, effort and producing more consistent results.


STUDY DESIGN, DOE, PRODUCT QUALITY AND STABILITY1:55 Chairperson’s RemarksRajiv Nayar, PhD, President, HTD Biosystems, Inc.

2:00 Protein Engineering Methods to Increase the in vivo Potency and Half-Life of Therapeutic ProteinsKrishna M.G. Mallela, PhD, Associate Professor of Biophysical Chemistry, Department of Pharmaceutical Sciences, Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado Anschutz Medical CampusIn this talk, we will discuss the protein engineering methods to increase the in vivo potency and half-life of therapeutic proteins. We will share some unpublished results where we were able to extend the in vivo potency of a pharmaceutical protein by 12-fold, and significantly increase its half-life.

2:30 Increasing Pharmaceutical Discovery and Development through a Rapid One Platform Approach: Getting More with Less without Losing QualityRajiv Nayar, PhD, President, HTD Biosystems, Inc.One of the major challenges facing drug developers is how to evaluate the “developability” of their biomolecules with respect to formulation and stability issues with very limited amounts of precious drug substance. An effective approach to rapidly identify an appropriate formulation in the minimum number of trials and getting the maximum amount of information using a multivariate response-surface DoE platform and high throughput analytics with principles of QbD is presented.

3:00 How to Utilize Design of Experiments (DoE) Principles for the Development of High Throughput, Robust Methods for the Assessment of Product QualityJeremy Springall PhD, Scientist I, Analytical Sciences, MedImmune, A member of the AstraZeneca GroupBeing able to generate accurate and precise analytical data to provide information on product quality in a timely manner is a great challenge facing analytical groups. By adopting a Design of Experiments (DoE) approach, we can overcome many hurdles facing the implementation and adoption of these high-throughput methods with the data generated being of comparable quality to that from longer lot release methods.


4:15 Sampling Statistics for Subvisible Particle Analysis of Drug ProductsMarina Gühlke, PhD, Analytical Scientist, Coriolis PharmaSubvisible particles analysis is required by the authorities for biopharmaceutical drug product development. Available sample volumes of biologics are often limited, and USP<787> allows light obscuration measurements at sample volumes of ~ 1 ml, in contrast to the traditional USP<788> (25 ml). However, careful consideration of analyzed sample volumes is necessary to obtain statistically relevant results, in particular for samples with low particle counts.

4:45 Mechanism for the Screening of Patchy Anisotropic Interactions in Protein Solutions Using Hydrophobic ExcipientsPrasad Sarangapani, PhD, Staff Scientist, Protein Biochemistry, Regeneron PharmaceuticalsIn this presentation, we will discuss and share results on mechanism for the screening of patchy anisotropic interactions in protein solutions using hydrophobic excipients.


6:00 - 8:30 Recommended Dinner Short Course*SC6B: Protein Aggregation: Mechanism, Characterization and Consequences* Separate registration required; click here for details.



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6TH ANNUAL Overcoming Formulation Challenges for Biopharmaceuticals DevelopmentHot Topics, Platform Technologies, Case Studies and Highlights from Industry


MONDAY, AUGUST 13


9:00 - 11:30 Recommended Short Course*SC3A: Application of New Analytical Tools in Protein Formulation Development and Subvisible Particle Characterization* Separate registration required; click here for details.


FORMULATION AND ANALYTICAL DEVELOPMENT OF NEW MODALITIES1:00 pm Chairperson’s Opening RemarksNicholas Warne, PhD, Senior Director, Pharmaceutical R&D, BioTherapeutics Pharmaceutical Sciences, Pfizer, Inc.

KEYNOTE PRESENTATIONS1:10 Delivering Flexible Dosage Forms for Innovative Modalities and an Evolving Development PlanNicholas Warne, PhD, Senior Director, Pharmaceutical R&D, BioTherapeutics Pharmaceutical Sciences, Pfizer, Inc.The development of biopharmaceutical processes and products is predicated on assumptions of dose range, time to customize the dosage form and market preferences. While seeking efficient approaches to screening compounds to assess developability, the increased complexity of biologics requires innovative approaches to formulation and dosage form development. The balance of platform processes to create capacity to provide innovative formulation solutions to novel modalities will be the focus of this presentation.

1:45 Challenges in Downstream Processing and Analytics of a Large Diversity of Molecules/Formats in the Discovery SettingJohn E. Harlan, PhD, Principle Research Scientist, Global Protein Sciences, AbbVieDownstream processing in support of Biologics Drug Discovery provides challenges that are distinct from those encountered in Development. Here we will discuss the approach we have taken to develop platform processes that can address the large diversity of molecules we encounter in the Discovery setting in the shortened timelines needed to facilitate the Discovery process, including the fit for purpose analytical approach we take, and automated processes we have developed.

2:15 A Holistic Approach to Formulation Development: From Cell Line to Drug Product DevelopmentSonal Saluja, PhD, Scientist II, Technical Development, Biogen

An end-to-end approach is needed for formulation development. Process impurities and heterogenous drug substance generated from upstream/downstream processes can negatively impact drug stability. This might not always be resolved by optimizing the formulation. Hence, a holistic development approach involving balancing yield, productivity, quality attributes and drug product stability is needed to guarantee success. This talk will highlight this concept using case studies.


3:15 Formulation Challenges in the Increasingly Complex Patent LandscapeJan Jezek, PhD, CSO, Research & Development, Arecor, Ltd.With growing competition in the market and more complex regulatory requirements, the demands for formulation of therapeutic proteins are increasing. In addition, the formulation patent landscape is becoming more complicated which further contributes to the challenge. This talk will describe several examples of successful formulation-related blocking patents as well as strategies for developing successful products in the increasingly complex patent landscape.

EXCIPIENT AND PRESERVATIVE ISSUES AND ALTERNATIVES TO POLYSORBATES3:45 Development of Parenteral Multidose Product – The Challenge of PreservativesHelen Sjögren, PhD, Principal Scientist, Global Pharmaceutical R&D, Ferring PharmaceuticalsIt is a regulatory requirement to prove the efficacy of the preservation system in a multidose product. The preservation efficacy is not only depending on the concentration of the preservative, but also on other components in the formulation. It has previously been shown that interaction between peptides and preservatives reduces the preservation efficacy. This has also been proven to be the case for various excipients as surfactants, buffers and stabilisers. The effect varies with both excipient and preservative used.

4:15 SELECTED POSTER PRESENTATION: Raman Spectroscopy to Detect Protein Structural Changes in the Solid StateLauren Fontana, PhD Candidate, Department of Pharmaceutical Sciences, University of ConnecticutA simple analysis of the lyophilized protein solid immediately after processing (requiring no reconstitution) that predicts stability would be ideal. FTIR is used to monitor secondary structural changes, but with limited prediction ability. Raman spectroscopy has been investigated to characterize solid state secondary and tertiary protein structure. Principal component analysis of Raman spectra can detect some of the more subtle structural changes that are not apparent using traditional data analysis.

4:30 Breakout DiscussionsThis session provides the opportunity to discuss a focused topic with peers from around the world in an open, collegial setting. Select from the list of topics available



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and join the moderated discussion to share ideas, gain insights, establish collaborations or commiserate about persistent challenges. Then continue the discussion as you head into the lively exhibit hall for information about the latest technologies.

Product Differentiation Through Innovative FormulationModerator: Jan Jezek, PhD, CSO, Research & Development, Arecor, Ltd.• How to deal with an increasingly complex formulation patent landscape• Need for product differentiation in the increasingly competitive biologics

market• Enabling successful products through innovative formulation

How to Incorporate Excipient Quality and Specification Limits in Formulation Robustness StudiesModerator: Helen Sjögren, PhD, Principal Scientist, Global Pharmaceutical R&D, Ferring Pharmaceuticals• Surfactants, how to control quality, levels and stability in drug product –

regulatory requirements• Strategies for in-use stability, focus on infusion products with preparation

steps before administration

Screening Methods That Are Most Indicative of Final Formulation Parameter and Most Useful in Formulation Development and SelectionModerator: John E. Harlan, PhD, Principal Research Scientist, Global Protein Sciences, AbbVie• What screening method(s) are the most predictive of formulation stability?• Are there new methods/instruments that have the potential to be a “step

change” in the way formulation screening is conducted?• How do you probe high concentration behavior with limited sample available

for screening?

Understanding the Role and Effect of Buffers in Biopharmaceutical Formulation DevelopmentModerator: Shreya Kulkarni, PhD Candidate, Pharmaceutical Sciences, University of Connecticut• Importance of buffers in formulation design and stability.• Do buffer ions play a role other than buffering? (eg. by interacting with ionized

residues on the protein)• Is it possible to completely strip ions during buffer exchange? (Donnan

equilibrium)• What is the effect of buffers on drug product stability?


7:00 End of Day

TUESDAY, AUGUST 14


POLYSORBATE ISSUES AND NEW EXCIPIENTS7:55 Chairperson’s RemarksAndrea Hawe, PhD, CSO, Coriolis Pharma Research GmbH

8:00 Analytical Strategy for Polysorbate Degradation in Biopharmaceutical ProductsAndrea Hawe, PhD, CSO, Coriolis Pharma Research GmbHSetting up an appropriate analytical strategy to characterize polysorbate and degradation products thereof, is crucial for the development of polysorbate-containing drug products. The talk will focus on analytical methods for soluble PS species, e.g., LC-CAD, LC-MS, as well as polysorbate-related particles, and discuss the phase-appropriate application of the different methods. Case studies on polysorbate degradation relevant for biopharmaceutical formulations will be presented to show the application of the different methods.

8:30 A Case Study utilizing EDTA as a Stabilizing Excipient for Monoclonal Antibody Oxidation and Polysorbate 80 DegradationMelissa Yarbrough, MS, Scientist, Pharmaceutical Research and Development, Pfizer, Inc. Polysorbates are frequently utilized in biotherapeutic formulations. Interest in assessing their stability, in particular the impact of their degradation products, on the stability of therapeutic proteins have been steadily growing in the past decade. A case study of a monoclonal antibody formulation that demonstrated a simultaneous loss of polysorbate and an increase in methionine oxidation will be presented. Further investigation demonstrated that the inclusion of EDTA decreased methionine oxidation and prevented surfactant degradation.

9:00 Oxidative Degradation of Polysorbate: Novel Mechanisms and Effects of Formulation BuffersIndira Prajapati, PhD Candidate, Schoeneich Lab, Department of Pharmaceutical Chemistry, The University of Kansas ON BEHALF OFChristian Schoneich, PhD, Distinguished Professor and Chair, Pharmaceutical Chemistry, University of KansasProtein formulations frequently contain polysorbate as surfactant. The oxidative degradation of polysorbate leads to multiple products including free fatty acids and various polyoxyethylene esters. We report here on a new pathway to free fatty acid generation, as well as reactions of formulation buffers (e.g., histidine and acetate), which both inhibit and promote polysorbate oxidation.

Sponsored by9:30 Using Metabolomics to Understand Genetically Determined and Environmental Cell Culture NeedsBrian Keppler, PhD, Associate Director, Project Management, MetabolonWe will describe how metabolomics can be applied across the bioprocess lifecycle from process development to process monitoring can yield biological insight to facilitate the development, implementation, and execution of robust and



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reproducible bioprocesses. In addition,we will discuss the role of biochemistry in immunometabolism and in development optimization of immunotherapies.


10:30 Just below the Surface: New Excipient Alternatives to Polysorbate SurfactantsPhilip Wuthrich, Senior Scientist, Research, Research & Development, ReForm BiologicsCommonly used polysorbate surfactants are prone to chemical degradation, compromising formulation storage stability. Polysorbates also create processing issues, because the micelles they form are similar in size to antibodies. Here we present data for a new alternative excipient that can match polysorbate performance at comparable addition levels without the chemical instability and processing limitations associated with polysorbates.

NEW DEVELOPMENTS IN LYOPHILIZATION AND ALTERNATIVE DRYING TECHNIQUES11:00 Spray Drying of Biopharmaceuticals for Mitigation of Cold Chain Requirements – A Comprehensive Monoclonal Antibody Case StudySonia Bedi, PhD, Investigator, Biopharm Product Sciences, Biopharm R&D, GSKThe present work showcases a comprehensive approach to spray drying a monoclonal antibody with the objective of mitigating cold chain and driving the technology of spray drying biologics one step closer to being successfully industrialized. The ability to powder fill individual unit doses with high precision was also investigated. The topic is very pertinent to the current biopharmaceutical industry i.e. solid state stabilization of proteins.

11:30 Mass Spectrometry for Equipment and Process Monitoring during Freeze DryingJason Stewart, BS, Senior Associate Scientist, Pharmaceutical Research and Development, Pfizer, Inc.Mass spectrometry has emerged as a key technology for the detection of trace levels of silicone oil in freeze drying chamber. With the capability of measuring low molecular mass gasses such as water and nitrogen, mass spectrometry positions itself to be a powerful tool for not just equipment monitoring but the entire drying process.

12:00 pm SELECTED POSTER PRESENTATION: Improvement in Thermal Stability of Antibodies and ADCs with Rationally Chosen FormulationsJunia Dushime, Senior Research Associate, Magenta TherapeuticsIn this case study, an antibody presented with undesirable thermal instability as both an unmodified antibody and when conjugated to produce an ADC. A high throughput formulation screening was executed to study the impact of numerous buffers over a pH range. A clear improvement in thermal stability was observed when both the antibody and ADC were subjected to thermal stress in non-phosphate buffers. Thermal stability was confirmed by visual examination and relevant analytical assays such as turbidity, SEC, HIC, DSF, and CE-IEF. Here, we will describe our early formulation approach and development of appropriate analytical methods and present our results.

Sponsored by12:30 Luncheon Presentation: Rip Through Formulation Prep with a Junior, a Lunatic and an Uncle Joe Barco, PhD, Senior Director, Marketing, Unchained LabsIdentifying formulations for biologics is a painful process Automated buffer exchange coupled with volume control can streamline the buffer exchange process Lower volume analytic measurements provides a means to get to key formulations faster and more effectively Combining automated buffer exchange with low sample volume analytics increases throughput and reduces sample burdens for discovery


DEVELOPABILITY, ROBUSTNESS, AND SCALE-UP1:55 Chairperson’s RemarksAtul Saluja, PhD, Associate Director, Global Pharmaceutical Development Biologics, Sanofi

2:00 Considerations for Designing Robust Clinical In-Use StudiesAtul Saluja, PhD, Associate Director, Global Pharmaceutical Development Biologics, SanofiClinical in-use stability studies are critical in assigning an in-use period and in defining necessary study components to enable administration of an active drug product in a safe and efficacious manner. Designing these studies is often challenging given the multitude of factors that need to be considered. This talk will focus on various clinical, CMC, regulatory, analytical, and logistical aspects that must be considered in order to design robust clinical in-use studies.

2:30 An Intercompany Perspective on Biopharmaceutical Drug Product Robustness StudiesTanvir Tabish, PhD, Head, Drug Product Development for Gene Therapy, Device and Combination Products, ShireDrug Product (DP) robustness studies ensure a product meets quality standards when formulation and process parameters are within established ranges. The BioPhorum Development Group (BPDG) authored a commentary on DP robustness (S. Morar-Mitrica, et al., J.Pharm.Sci. (2018), 107: 529-542) focused on the current scope, design and execution of biopharmaceutical DP robustness studies. Common themes were identified with regard to the use of DP robustness studies, with integrated case studies.

3:00 Early Stage Physicochemical Characterization of Novel Format ADCsPamela Thompson, PhD, Senior Scientist II, ADC Preformulation, NBE Development, AbbVieBridging early drug discovery and downstream development of novel format ADCs offers a challenging task in identifying the optimal biophysical techniques to investigate their developmental risk and physicochemical stability. This presentation will focus on our ADC Preformulation approach which implements an efficient and robust screening funnel platform for ADC candidate selection.




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4:15 Preventing Photo-Induced Degradation of an ADCAmit Gangar, PhD, Scientist II, Analytical and Pharmaceutical Sciences, ImmunoGen, Inc.Stabilizing antibody-drug conjugates (ADCs) during manufacturing and storage poses several challenges to the formulation development scientists. Formulation optimization is further complicated for a photosensitive ADC and requires additional excipient screening studies to ensure stability during manufacturing and drug administration. A case study focused on addressing the light induced instability of an ADC will be discussed in this presentation.

4:45 Effect of Light Source and UV Quotient on Monoclonal Antibodies Stability during Manufacturing and StorageHaresh T. More, PhD, Research Investigator II, Parenteral Science and Technology, Bristol-Myers SquibbBiologics molecules are known to be photosensitive, especially to UV light, and photo-induced degradation of proteins can occur at any point from drug substance and drug product manufacturing up to final delivery to patient. During this entire process, the biologics drug product is exposed to different types of lights that may include some degree of UV in the 200 - 400 nm range. In this study, we evaluated different light sources for their UV quotient and light fixture UV-filter capabilities. The effect of UV leakage on aggregation was explored under these lights.





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7TH ANNUAL

High-Concentration Protein FormulationsNew Technologies, Devices, Analytics, Case Studies and New Research Highlights


FOR DETECTION, CHARACTERIZATION AND CONTROL OF IMPURITIES IN BIOLOGICS

AGENDA taking place Thursday, August 16 and Friday, August 17, please click here.

TUESDAY, AUGUST 14




MANUFACTURING AND PROCESS CHALLENGES8:05 Chairperson’s RemarksJean-René Authelin, PhD, Global Head of Pharmaceutical Engineering, Pharmaceutical Sciences, Sanofi France

8:15 KEYNOTE PRESENTATION: Challenges in Industrial-Scale Fill & Finish Operations for High-Concentration Proteins FormulationsJean-René Authelin, PhD, Global Head of Pharmaceutical Engineering, Pharmaceutical Sciences, Sanofi FranceHigh-concentration protein formulations are more and more required to administer proteins, especially mAbs. However, due to their high viscosity, propensity to aggregate or to form gel by drying, there are many hurdles to develop a robust commercial fill & finish process. The role of product development studies, which will be shared during this talk, is to anticipate the main difficulties and to adapt the process to the product by setting up a de-risking methodology, based on downscaled models and the use of well-designed surrogates.

9:00 Time-Pressure Filling of Concentrated mAbs Solutions: Understanding the Temperature Impact to Better Control the Filling Step (A Case Study)Charlotte Pellet, PhD, Pharmaceutical Sciences Operations, Sanofi, FranceFill & finish process for parenteral products includes a filling step crucial to obtain accurate and reproducible doses in syringes. It becomes a great technical challenge for concentrated mAbs solutions of high viscosity, strongly dependent on concentration and temperature. Understanding both rheology and complex machine automatism is the key. I will present a study case in which we rationalized and modeled the impact of very small temperature effects on filling accuracy.

9:30 PANEL DISCUSSION: Managing the Manufacturing and Process Challenges Posed by High Viscosity• Challenges in scaling up high-concentration protein formulations• New techniques and tools for processing at high concentrations• Share your own experiences and problems when processing high-concentration formulations and how the problem was approached and solved• What’s in the future?

Moderator:Thomas Laue, PhD, Professor Emeritus, Molecular, Cellular and Biomedical Sciences, University of New HampshirePanelists:Jean-René Authelin, PhD, Global Head of Pharmaceutical Engineering, Pharmaceutical Sciences, Sanofi FranceDeborah Bitterfield, PhD, CEO and Founder, Lindy Biosciences, LLCRobin Bogner, PhD, Professor, Department of Pharmaceutical Sciences, School of Pharmacy, University of ConnecticutQahera Munaim, Process Development Senior Associate, Pivotal Drug Product, Amgen


LYOPHILIZATION & ALTERNATIVE DRYING TECHNIQUES FOR HIGH-CONCENTRATION PROTEIN FORMULATIONS10:45 Single Step Drying: Challenges and Considerations for Freeze-Drying High Concentration Protein FormulationsSwapnil Pansare, Associate Scientist II, Dosage Form Design & Development, MedImmuneLyophilization process optimization enables reduced cost and increased Drug Product manufacturing throughput. Typically, drying is performed below the collapse temperature to produce an elegant product appearance. Here, drying above the collapse temperature was performed in a single step by combining primary and secondary steps, without impacting product appearance. A case study is presented to assess the impact of single step drying on product quality, process efficiency and scale up.

11:15 Factors Governing the Reconstitution Time of High-Concentration Lyophilized Protein FormulationsRobin Bogner, PhD, Professor, Department of Pharmaceutical Sciences, School of Pharmacy, University of ConnecticutCo-Presenter: Shreya Kulkarni, PhD Candidate, Pharmaceutical Sciences, University of ConnecticutA major challenge to the development of high-concentration lyophilized protein formulations is slow and variable reconstitution times, sometimes exceeding 1 hour. Long reconstitution times are especially undesirable for self-administered products. As biosimilars and additional protein products come to market, reduced reconstitution time is expected to be a differentiating factor. The overall goal of our research is to offer a fundamental understanding of the variables governing reconstitution in both amorphous and partially crystalline freeze-dried cakes and to identify the key cake attributes predictive of reconstitution time.

Sponsored by11:45 Delivery of High-Concentration Protein Suspensions Deborah Bitterfield, PhD, CEO and Founder, Lindy Biosciences, LLC



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1:00 Session Break

PREDICTION & MEASUREMENT OF PROTEIN-PROTEIN INTERACTIONS, VISCOSITY & SOLUBILITY1:45 Chairperson’s RemarksRobin Bogner, PhD, Professor, Department of Pharmaceutical Sciences, School of Pharmacy, University of Connecticut

1:50 Biotherapeutic Protein Size and Charge and Colloidal StabilityThomas Laue, PhD, Professor Emeritus, Molecular, Cellular and Biomedical Sciences, University of New HampshireGood colloidal stability is required to develop low-viscosity, stable high-concentration protein solutions. Charge and charge distribution are major contributors to protein solubility and rheology. Data are presented to show that the amount of charge needed for good colloidal solubility increases with the square of the Stokes radius. This means that a bispecific mAb of 7 nm radius will require nearly twice the charge to achieve the same solubility as a 5 nm mAb. Protein charge cannot be calculated, and importance of measuring the charge will be emphasized.

2:20 Solubility Theory and Relevance to Protein-Protein Interactions Underlying Viscosity in High-Concentration Protein FormulationsQahera Munaim, Process Development Senior Associate, Pivotal Drug Product, AmgenAt high protein concentration, many proteins are viscous, resulting in formulations that cannot be administered via parenteral injection. In this work, we demonstrate that for water miscible solvents, common properties of these solvents may be important in minimizing viscosity. These properties include dispersion forces, polar forces and hydrogen bonding. A case example will show how evaluating solubility parameter space is relevant in mitigating the effects of solution viscosity at high concentration.

2:50 Applying Modified Colloidal Theories to Model Protein Solutions from Low to High Protein ConcentrationsCesar Calero-Rubio, PhD, Scientist, Formulation Development, Global Pharmaceutical Development Biologics, SanofiSeveral experimental and computational methodologies are combined at low protein concentrations (c2) to model protein self-interactions. Scattering techniques are used to measure self-interaction parameters of protein solutions as a function of solution formulation. Molecular scale simulations are used to predict protein self-interactions at high c2 based on low-c2 measurements. The results show the viability and limitations of using simplified molecular models to predict protein self-interactions.

3:20 Thermodynamics of Osmolyte-mAb Interaction in Aqueous SolutionsChaitanya Sudrik, Postdoctoral Associate, Chemical Engineering, Massachusetts Institute of TechnologyWe have investigated via vapor pressure osmometry the interactions of osmolytes in 3 antibodies with different properties. The transfer free energy (0.5-0.9 kJ/mol per 1000 Ao2 of protein-water interface, for 200 mM osmolyte) follows the trend: trehalose and sucrose> sorbitol and is not affected by a change in solution pH from 5.5 to 7.2 or change in the buffer from 25 mM Na-acetate to 25 mM Histidine.HCl.




7:30 End of Day

THURSDAY, AUGUST 16


PREDICTION & MEASUREMENT OF PROTEIN-PROTEIN INTERACTIONS, VISCOSITY & SOLUBILITY (CONT.)8:25 Chairperson’s RemarksJan Jezek, PhD, CSO, Research & Development, Arecor, Ltd.

8:30 Weak IgG Self- and Hetero-Association Characterized by Fluorescence Analytical UltracentrifugationDanlin Yang, PhD, Senior Scientist, Biotherapeutics Discovery, Boehringer IngelheimWeak protein-protein interactions may be important to binding cooperativity. A panel of 7 fluorescently labelled tracer IgG antibodies, differing in variable (V) and constant (C) region sequences, were sedimented in increasing concentrations of unlabeled IgGs of identical, similar, and different backgrounds by analytical ultracentrifuge fluorescence detection. Weak IgG:IgG attractive interactions were detected and characterized by global analysis of the hydrodynamic nonideality coefficient, ks.

FORMULATION DESIGN, DRUG DELIVERY AND SMART DEVICES9:00 Successful High-Concentration Products – Creative Formulation and Device ChoiceJan Jezek, PhD, CSO, Research & Development, Arecor, Ltd.Developing stable, low-viscosity therapeutic protein products is extremely challenging and requires a number of good decisions to be made along the development



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7TH ANNUAL





process with respect to the product concept, formulation, delivery device. The talk will describe innovative formulation approaches enabling differentiated product concepts, as well as examples of intellectual property considerations that have to be taken into account during the development.

9:30 SELECTED POSTER PRESENTATION: Liquid-Liquid Equilibrium of a Monoclonal Antibody in Terms of Protein-Protein InteractionsNicole Sibanda, School of Chemical Engineering and Analytical Science, University of ManchesterThe aim of this work is to develop methods for predicting concentrated solution thermodynamic properties, in particular liquid-liquid phase separation from dilute solution measurements of protein-protein interactions (PPIs). We have characterized the phase diagram of a mAb in terms of the second virial coefficient at room temperature by varying the ionic strength of the solution. In addition, dynamic and static light scattering and sedimentation velocity experiments have been used to characterize the behavior along sub-critical isotherms at protein concentrations up to the dilute branch of the binodal. This data is then used to propose a simplified colloidal model that can be used to extrapolate the dilute solution measurements to predict the phase behavior.


10:45 Identification and Characterization of Excipients and Excipient Mixtures Based on Thermodynamic Models and Advanced AnalyticsChristoph Brandenbusch, PhD, Group Leader, Department of Biochemical and Chemical Engineering, Laboratory of Thermodynamics, Technical University of DortmundClassically, excipients and excipient mixtures are identified based on heuristic or “trial-and-error” based approaches. However, it is unlikely that this results in optimal formulations. It is thus desired to develop a physically-sound method to identify excipients and excipient mixtures based on modeling/predicting the intermolecular interactions in solution. This allows to access the suitability of multi-excipient systems and to determine the optimal excipient mixture in an early stage, circumventing cost-intensive screening methods.

11:15 Engineering Proteins for Long-acting Ocular DeliveryDevin Tesar, PhD, Scientist, Drug Delivery, Genentech, Inc.Ocular delivery of protein therapeutics often requires favorable viscosity properties to support high concentration formulations. Using structure-based design we generated high-affinity mutants of a backup Fab which exhibited superior viscosity properties but inferior target binding and inhibition as compared to the lead candidate. Two of these mutants, FM1 and FM2, exhibit binding and target inhibition equal or superior to that of the lead molecule, while retaining the superior viscosity profile.

11:45 A Virtual Formulation Screening Tool for High Concentration Antibodies: Formulations with Reduced ViscositySarah Altinoglu, PhD, Post-Doctoral Scientist, Bioproduct Research and Development, Eli Lilly and CompanyDevelopment of high concentration antibody formulations (>150mg/ml) require consideration of product viscosity, leading to additional conditions to evaluate and compounding the already high material and time requirements to develop the proper formulation. A virtual tool to aid in screening out formulation conditions with the highest likelihood of high viscosity was built based on the correlation analysis of antibody molecular properties and formulation viscosity responses.




6:00 - 9:00 Recommended Dinner Short Course*SC11C: Development of Innovative and Competitive Biologic Formulations in Complex Patent Landscape* Separate registration required; click here for details.



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Cell Therapy The FDA’s approval of Novartis’s Kymirah and Gilead’s Yescarta has propelled cell-based therapies into the mainstream. However, manufacturing and characterizing these therapies at scale remains the next big challenge.

The Cell Therapy CMC and Manufacturing stream comprises two back-to-back conferences exploring the critical challenges facing the analysis and production of cell therapies with an emphasis on overcoming the practical challenges. Topics include international regulations, CMC strategies, characterization, automation in analytics and manufacturing, scaling up CRISPR-based CAR Ts, assay development, technology development and product release.

AUGUST 13-14

AGENDA Cell Therapy CMC and Analytics

AUGUST 15-16

AGENDA Cell Therapy Manufacturing

AUGUST 16-17

AGENDA Design of Experiments for Cell and Gene Therapies

2018 CELL THERAPY TRACKS



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3RD ANNUAL

Cell Therapy CMC and AnalyticsRegulation, Characterization and Control of Cell-Based Therapies

Cell Therapy

MONDAY, AUGUST 13


9:00 - 11:30 Recommended Short Course*SC4A: Lessons Learned from the First 17 ATMPs Submitted to the EMA* Separate registration required; click here for details.


REGULATORY REQUIREMENTS1:00 pm Chairperson’s Opening RemarksFouad Atouf, PhD, Vice President, Global Biologics, USP

1:10 Cell Therapy Product Manufacturing ConsiderationsBernadette Keane, PhD, Principal, Keane ConsultancyCell therapies are substantially more complex than small molecule or biological approaches to medicine. This complexity poses challenges for both academic groups and companies developing cell therapies, as well as for regulators seeking to oversee this growing area of medicine. In this interactive session, we will discuss some of the common challenges and lessons learned along the way and explore how collaborations between industry and the regulators can help lead to successful translation and commercialization of cell therapies.

1:45 Regulatory Aspects of Manufacturing and Control of Genetically Modified CellsMatthias Renner, PhD, Scientist, Federal Institute for Vaccines and Biomedicines, Paul Ehrlich InstituteIn respect to manufacturing and quality control, genetically modified cells are considered to be most complex medicinal products. Regulatory aspects considering the fundamental steps in manufacturing and control of these medicinal products will be presented, and the regulatory framework for these products which are classified in the EU as advanced therapy medicinal products and are regulated centrally by the European Commission and the European Medicines Agency will be given.

2:15 Standards and Best Practices Applicable for Advanced TherapiesFouad Atouf, PhD, Vice President, Global Biologics, USPQualification of raw materials used in the manufacturing of cellular therapies, requires the use of risk assessment strategies to categorize the critical components of a manufacturing process. In addition to cell culture supplements, excipients and other formulation’s components must meet the required quality to ensure consistency in manufacturing and subsequently the quality and safety of finished cell therapy products. This presentation will discuss the critical strategies facing the development of cell therapies as per USP recommendations.


CMC STRATEGIES FOR CELL THERAPIES3:15 FDA’s CMC Review of a Cell TherapeuticsWilliam Lee, PhD, Vice President, Regulatory Affairs, Cato ResearchThis talk will focus on two case studies. The first case study will discuss FDA’s CMC issues with a stem cell therapy that were raised at a pre-IND meeting. The second case study will discuss FDA’s CMC questions for an investigational cell therapy during the pre-IND meeting and then subsequently during the IND 30-day review after initial IND submission.

3:45 PANEL DISCUSSION: Regulatory and CMC Strategies for Cell Therapies• Most common questions asked by companies/ regulators• Lessons learnt from recently approved products• Challenges around gene-edited cell therapies• Preparing for IND – What are the priorities, preclinical packages?Moderator:Fouad Atouf, PhD, Vice President, Global Biologics, USPPanelists:Bernadette Keane, PhD, Principal, Keane ConsultancyMatthias Renner, PhD, Scientist, Federal Institute for Vaccines and Biomedicines, Paul Ehrlich Institute




7:00 End of Day

TUESDAY, AUGUST 14


CELL COUNTING AND PRODUCT CHARACTERIZATION7:55 Chairperson’s RemarksChristopher Bravery, PhD, Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd.



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8:00 Tools and Strategies to Improve Confidence in Cell Counting MeasurementsLaura Pierce, PhD, Biomedical Engineer, Biosystems and Biomaterials Division, Biomaterials Group, NISTCell count, a routinely performed fundamental measurement in the biosciences, underpins key decisions in the manufacturing, commercialization, and release of cell-based therapies. The industry seeks tools to gain greater confidence in cell counting measurements. NIST, in collaboration with industry and other government agencies, is developing strategies to provide fit-for-purpose measurement assurance for cell counting, in the forms of written standards, experimental designs, and control materials.

8:30 Towards Rigor and Reproducibility in Single Cell Analytics for Therapeutic Cell ProductsRuud Hulspas, PhD, Independent Consultant, Cellular Technologies Bioconsulting, LLCCell characterization occurs at multiple checkpoints of the manufacturing process and is particularly important at product release. In contrast to bulk analysis, single cell analysis offers detailed information about the composition of therapeutic cell products. Flow cytometry provides rapid analysis of millions of cells for up to 30 cellular parameters, simultaneously measured on individual cells, but it can also be difficult, unreliable and expensive. Existing expertise and new developments are applied for successful integration in cell therapy.

9:00 Assays in Purification of Targeted Cell PopulationsPatricia Rogers, Manager, Cell Sorting Capability, Broad Institute of MIT and HarvardCell purification of targeted populations is necessary when specific populations are needed for downstream applications. Instrumentation needed in order to purify cell populations is often complicated and hard to standardize. Also, there is biological variation between samples that needs to be accounted for. This presentation will focus on understanding the key areas for variation and developing tools to standardize each part of the assay in order to provide reproducible results.

9:30 Developing 21st Century Rapid Sterility Testing for Cell-Based TherapiesJacqueline Wolfrum, PhD, Associate Director, Biomanufacturing Program (BioMAN), Center for Biomedical Innovation, Massachusetts Institute of Technology


POTENCY, AUTOMATED ANALYTICS AND RAPID RELEASE10:30 Potency Assay Development and Qualification for B-Thalassemia and Sickle Cell Disease Autologous Gene Therapy Drug ProductsIlya Shestopalov, PhD, Senior Scientist, Cellular Process Characterization and Analytics, bluebird bioQuantitative potency assays were developed to demonstrate correction of b-thalassemia and sickle cell disease properties in an in vitro cell culture system. Potency was found to be specific to the beta-globin lentiviral vector and dependent on transduction efficiency of the autologous gene therapy drug product, demonstrating ability to reject sub-functional drug products. Considerations for assay development, qualification results, and redundancy to transduction efficiency methods will be discussed.

11:00 Product Release towards Rapid Quality ControlJunxia Wang, PhD., Director, Analytical Development, Mustang Bio, Inc.As “living drugs”, cell therapies pose unique analytical characterization challenges; these challenges extend from the research bench to the cGMP manufacturing setting. This talk will review common analytical methods for cell therapies, specifically CAR T- and TCR-based therapies, with a specific emphasis on the limitations of current methods and the move towards automated analytical methods.

11:30 PANEL DISCUSSION: Automation in Analytics and Manufacturing• Rapid release testing strategies – purity, rapid sterility testing, what does it mean? Which tests are necessary, which are not?• Automated/semi-automated analytics – regulatory considerations, options, issues• Adventitious agent testingModerator:Christopher Bravery, PhD, Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd.Panelists:Rodney Rietz, PhD, Senior Investigator, Exploratory Immuno-Oncology, NovartisJunxia Wang, PhD., Director, Analytical Development, Mustang Bio, Inc.Jacqueline Wolfrum, PhD, Associate Director, Biomanufacturing Program (BioMAN), Center for Biomedical Innovation, Massachusetts Institute of Technology

Sponsored by12:00 pm Single Cell Proteomics for Evaluating Function & Clinical Utility in Cell Therapy Sean Mackay, CEO, IsoPlexisBy capturing 40+ secreted proteins per single cell, thousands of cells at at time, researchers can determine the functional activity and strength of immune cells and cellular products. Using the IsoCode technology to define T-cell functional strength, Kite Pharma has used their pre-infusion product to predict objective response to CAR-T therapy. Isocode technology has also provided biomarker correlates to objective response in checkpoint inhibitor therapy - both from TILs and PBMC.



COMPARABILITY, STABILITY AND VECTOR ANALYSIS1:55 Chairperson’s RemarksScott R. Burger, MD, Principal, Advanced Cell & Gene Therapy, LLC

2:00 Assessment of Comparability of a Gene Therapy Drug Product after Manufacturing Site and Process ChangesStephen J. Duguay, PhD, Director, Cellular Process Characterization and Analytics, bluebird bioManufacturing site changes and process improvements were implemented during clinical evaluation of an autologous cell-based gene therapy. A risk based approach

3RD ANNUAL


Cell Therapy



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was used to rank the potential impact of the changes to product quality, safety and efficacy. Statistical evaluation of study results included equivalence testing, quality ranges and qualitative comparisons. The study design, results and conclusions will be discussed.

2:30 In vitro Product Characterization and Stability Studies of CAR T Cell TherapiesCarlotta Peticone, PhD, Senior Scientist I, Process Development, Autolus

3:00 How Much Information Do I Need to Present for the Vector of My Genetically Modified Cell Product?Christopher Bravery, PhD, Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd.How do regulatory agencies view the vector of a genetically modified cell? Does the vector need to be GMP; from what point in manufacture would this apply? Can I use a drug masterfile? How much information, including data, should I provide in the dossier for clinical trials; will this be different for approval?


4:15 Designing Strategies for Data-Driven Product CharacterizationMarc-Olivier Baradez, PhD, Lead Analytical Development Scientist, Cell and Gene Therapy CatapultIn the development of cell therapy products, the ability to characterize the product during its manufacture is becoming as valuable as the ability to control and optimize the process itself. This presentation will explain the current limitations and opportunities associated with such dual product/process characterization, and it will cover the principles behind a suitable data-driven strategy for efficient product/process development in the context of industrialization.

4:45 Big Data Strategies for Cell Therapy ManufacturingScott R. Burger, MD, Principal, Advanced Cell & Gene Therapy, LLCThe quest to retrieve, analyze, and leverage that data has become the new gold rush in life sciences. This presentation will discuss the role of big data in cell therapy process development, real time analytics and commercial scale manufacturing.


6:00 - 8:30 Recommended Dinner Short Course*SC10B: My Potency Assay Is Quantitative – But Is It Quantitative for Potency?* Separate registration required; click here for details.

3RD ANNUAL


Cell Therapy



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5TH ANNUAL

Cell Therapy ManufacturingCommercializing Cell-Based Therapies

Cell Therapy



COMMERCIALIZATION STRATEGIES8:05 Chairperson’s RemarksOhad Karnieli, PhD, MBA, CEO and Co-Founder, Atvio Biotech Ltd.; Chair, Process and Product Development Subcommittee, International Society of Cellular Therapies

8:15 KEYNOTE PRESENTATION: Manufacturing for Cell Therapies: Modeling the Decision on Make vs. BuyKnut Niss, PhD, CTO, Mustang BioAutologous cell therapies require intensive manufacturing resources and while establishing a new manufacturing facility can be costly, it is critical to compare these costs to the ones required to manufacture at a CMO. In this respect, establishing a comprehensive model that looks at all aspects can serve as a critical tool to make this decision. This presentation will go through the modeling process with a look at several different cell therapy platforms.

9:00 Manufacturing Complex Therapies - In-House, Outside or a Mix?Devyn Smith, PhD, Chief Strategy Officer, Strategy & Ops, SigilonMaking a decision on where and how to manufacture a therapy is one of the most important decisions a company will make. The decision has impact on both scientific and clinical strategies, as well as fundraising/capital strategies. Criteria for considering how to make the most informed decision will be reviewed with case studies.

9:30 Technical Transfer and Facility Start-Up of CAR T ManufacturingBrian Murphy, PhD, Director, Bioprocess Development, Celgene


CAR-T MANUFACTURING10:45 Scalability Challenges with Molecular Switch Technologies for CAR TAlan Smith, PhD, Executive Vice President, Technical Operations, BellicumSuccessful manufacture of a patient-specific CAR-T cell product is multifaceted and complex. Areas which need to be successfully managed and solved include logistics from the starting cell collection through product delivery, vendor qualification and management and consistent sourcing of appropriate materials and equipment. Process reliability and product consistency are also challenges due to differences in patient age, genetics and disease treatments which vary substantially across the patient population.

11:15 Development of the Manufacturing Process of CAR T Cell TherapiesPrzemyslaw Kruczek, PhD, Senior Scientist, Autolus

Sponsored by11:45 Cell Counting Method Development Process for CAR T Cell ManufacturingLeo Chan, Manager, Technology Research & Development, Nexcelom Bioscience, LLCCell counting is highly important for all stages of cellular therapy development. The need for an accurate and consistent cell counting method is crucial for manufacturing under GMP/GLP conditions. Utilizing both bright field and fluorescence imaging, Cellometer automated cell counters can provide rapid and accurate measurement of cell concentration and viability at any stage: from CAR T product development to manufacturing.

Sponsored by12:15 pm Luncheon Presentation: Corning Solutions for Optimizing Cell Culture Environment in Cell Therapy ApplicationsKeith R. Olson, PhD, Global Director, Business Operations, Life Sciences, Corning IncorporatedOptimal cell culture conditions are critical to manufacture the highest quality cell therapy products. As a leader in cell culture, Corning offers complete and customizable cell culture environments suited for anchorage-dependent or suspension cells that are easily optimized to generate the highest quality cellular products. In this presentation, we will discuss the importance of building an optimal culture environment using various Corning platforms to suit your stage of cell therapy research. We will present Corning’s customizable vessels and media for cell therapy applications, as well as advanced cell culture surfaces, including synthetic and biological surfaces and extracellular matrices. In addition, we will feature Corning’s Dissolvable Microcarrier technology for scalable expansion of anchorage-dependent cells and discuss how it can simplify your cell harvest and separation processes.

1:00 Session Break

AUTOMATION, CLOSED SYSTEMS AND BIOSENSORS1:45 Chairperson’s RemarksKnut Niss, PhD, CTO, Mustang Bio

1:50 Transforming Cell Therapy with Gene Editing: The Case of “Off-the-Shelf” Engineered CAR-TDavid Sourdive, PhD, Executive Vice President, Technical Operations, CellectisGene-editing has enabled off-the-shelf allogeneic CAR-T product candidates to reach the clinic. It is also endowing engineered cells with multiple new features, enhancing their capabilities and functions to better address cancer. Hindsight in industrializing these immuno-oncology products and the human clinical experience with the first cases in ongoing trials signal practical avenues for their further deployment and shed light on the transformative role they will play in the anti-cancer arsenal.

2:20 Next-Generation CAR T Manufacturing, Bedside Automated Manufacturing, No Longer a DreamOhad Karnieli, PhD, MBA, CEO and Co-Founder, Atvio Biotech Ltd.; Chair, Process and Product Development Subcommittee, International Society of Cellular Therapies



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5TH ANNUAL

Cell Therapy ManufacturingCommercializing Cell-Based Therapies

Cell Therapy

Several approaches have been taken in recent years to automate the system, from automating unit operations to innovative end-to-end solutions. The talk will cover the available new technologies and future trends and will present a case study of a CAR T therapy end-to-end automated process called ADVA.

2:50 Low Cost Closed Systems for the Research and Preclinical StageShashi Murthy, PhD, Professor, Chemical Engineering, Northeastern UniversityMy major focus is building low cost automation platforms that can be easily adopted in preclinical and research settings. A major advantage of embedding automation early on is that transition to automation for clinical trials is considerably simplified and furthermore automation improves robustness of the early stage work. My presentation will discuss the above issues, plus topics like pros and cons of closed system, sensors, within the context of manufacturing dendritic cell-based therapies.

3:20 Application of Biosensors Technologies for Real Time Process ControlDamian Marshall, PhD, Head, Analytical Development, Non-Clinical Operations, Cell & Gene Therapy Catapult UKDeveloping reliable, cost-effective processes for cell and gene therapy manufacture is a significant challenge. For autologous products, this challenge can be even greater due to the variability of the patient specific starting material and the lack of real-time process data. In this presentation we will show how optical biosensors can be applied for process monitoring to track cell behavior and gain higher level process control.




7:30 End of Day

THURSDAY, AUGUST 16


SCALE-UP AND MANUFACTURE8:25 Chairperson’s RemarksOhad Karnieli, PhD, MBA, CEO and Co-Founder, Atvio Biotech Ltd.; Chair, Process and Product Development Subcommittee, International Society of Cellular Therapies

8:30 Overcoming Challenges in Advancing Allogeneic Cell Therapy beyond Phase IITehila Sonnenfeld, PhD, Director, Manufacturing & Technology Transfer, Immunovative TherapiesAllogeneic cell therapy products have the potential for achieving economies of scale. As biological products, these therapies are licensed together with the manufacturing processes and facilities. Therefore, careful planning is required in order to assure

manufacturing development occurs in parallel with the clinical development. This presentation will discuss our experiences in developing a scalable, economical manufacturing process for an allogeneic immune cell therapy for cancer.

9:00 Process Development: Lessons Learned from Virus-Specific T cellsAdrian Gee, PhD, Professor, Pediatrics-Hem-Oncology Cell & Gene, Baylor College of Medicine



BEYOND CAR-T: MANUFACTURING TILS AND T CELLS10:45 Scaling Up Cell Therapy Products in an Academic Cell Therapy FacilityPatrick J. Hanley, PhD, Laboratory Facility Director, Cellular Therapy and Stem Cell Processing, Program for Cell Enhancement and Technologies for Immunotherapy, Division of Blood and Marrow Transplantation, Children’s National Health SystemThis presentation will focus on our experience translating cellular therapies from a basic science discovery at the bench into a Phase I clinical trial. Included in this presentation will be how to engage all members of the team, from clinicians to the manufacturing team, and design a system to best meet the needs of the process. These items include reagent qualification, cell selection, staffing, and product testing.

11:15 Leveraging Academic and Commercial Cell Therapy Manufacturing Organizations to Facilitate Accelerated FDA ApprovalLinda L. Kelley, PhD, Senior Member, Director, Cell Therapy Facility, Moffitt Cancer CenterWe have embraced an academic and industry partnership to facilitate multicenter clinical trials using Tumor Infiltrating Lymphocytes (TIL) for solid tumors. Through a combination of process improvements intended to shorten product manufacturing time, as well as innovative implementation of GMP practices, we have maximized the contribution of academic and contract manufacturing organizations to support multiple ongoing trials intended to receive Fast Track FDA approval for a new cellular therapy.

11:45 Manufacturing Strategy for Chemotherapy-Resistant γδ T Cells for Treatment of GlioblastomaLawrence Lamb, PhD, Clinical Laboratory Immunologist, Professor of Medicine, The University of Alabama at Birmingham; Co-Founder, IncysusTMZ-resistant γδ T cells, injected in the post-resection tumor bed simultaneously with intravenous TMZ therapy, provide an active response to GBM when the tumor is in its most vulnerable state. We will discuss manufacturing operations for γδ T cell manufacturing and MGMT gene modification for our Phase I clinical trial that will begin accrual Summer 2018.



1:55 End of ConferenceA Division of Cambridge Innovation Institute


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Gene Therapy The Gene Therapy industry has undergone rapid advances over the last twelve months with the first gene therapy approved for sale in the US – Spark’s Luxturna, at the end of 2017. They don’t come cheap and companies are under incredible pressure to develop well defined, scalable therapies.

The Gene Therapy CMC and Bioproduction stream features two back-to-back conferences looking at the critical challenges facing the production, characterization and quality control of vector based gene therapies, with dedicated sessions on AAV and lentivirus-based platforms as well as oncolytic viruses, CRISPR and other gene editing therapies. Topics include potency assay development, stability, comparability and quality control, large-scale AAV and lentivirus process development, formulation, scale-up, product lifecycle management, industrialization, facility design and regulatory compliance.

AUGUST 15-16

AGENDA Gene Therapy CMC & Analytics

AUGUST 16-17

AGENDA Gene Therapy Manufacturing

2018 GENE THERAPY TRACKS



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2ND ANNUAL

Gene Therapy CMC and AnalyticsRegulation, Characterization and Control of Gene Therapies

Gene Therapy



REGULATORY EXPECTATIONS AND NEW STANDARDS8:05 Chairperson’s RemarksMichael Kelly, PhD, Director, Asset Leadership, Gene Therapy, Biogen

8:15 KEYNOTE PRESENTATION: Development of the First World Health Organisation (WHO) International Standard for Potency and Safety Control of Lentiviral Gene TherapyYuan Zhao, PhD, Head of Gene Therapy Section, Division of Advanced Therapies, NIBSC/Medicines and Healthcare Products Regulatory AgencyChanges in production sites and manufacturing processes have become increasingly common, posing challenges to developers regarding reproducibility and comparability of results. Development of a first WHO International Standard for gene therapy, is especially important given the usually orphan nature of the diseases to be treated, hampering the comparison of cross-trial and cross-manufacturing results for the important lentiviral vector platform.

9:00 FEATURED PRESENTATION: Viral Vector Manufacturing and Control: Regulatory Considerations and ChallengesMatthias Renner, PhD, Scientist, Federal Institute for Vaccines and Biomedicines, Paul Ehrlich Institute

9:30 Analytical Testing of Plasmids for Triple Transfection AAV Production: The Challenge of Defining Critical Material AttributesLawrence Thompson, PhD, Principal Scientist, Analytical Research & Development, BioTherapeutics Pharm Sciences, PfizerThe link between plasmid quality attributes and AAV production is a gap in our current knowledge set. Specifications for plasmids are set based on historical information. As in “it has always worked when we set these criteria”. In this study, we seek to create a link between the plasmid quality as assessed by the current control strategy and the quantity/quality of AAV produced.


VECTOR ANALYTICS AND CHARACTERIZATION10:45 Phase Analytics for AAV CharacterizationDanielle McAnally, PhD, Associate Scientist, Quality Control, AGTC

11:15 Characterization of a Dimeric Transgene Isoform and Its Potential Impact on AAV Product QualityRoman Raim, PhD, Analytical Product Owner, Process Development and Technical Services, ShireThe undesirable outcome of Factor IX (FIX) gene therapy Phase I/II clinical trial with BAX 335 led to a development of second generation of FIX construct. The second generation was developed as a single stranded FIX vector with the same capsid, (AAV8). We investigated the vector sub-population with encapsidated dimeric DNA isoform, checking for its biological activity and evaluating if the degree of this sub-population can be successfully reduced in the final product in order to increase overall product homogeneity.

Sponsored by11:45 Analysis of Purity and Packaging of Gene Therapy Vectors and VLPs to Support Process Development Decisions Josefina Nilsson, Head, EM Services, Electron Microscopy Services, VironovaLearn how innovative transmission electron microscopy (TEM)-based technology in combination with image analysis allows automatic imaging as well as automatic viral particle quantification and classification. Case study demonstrating how this technology can be used to automatically quantify the portion of intact as well as disrupted, empty AAV particles. Case study demonstrating how automated TEM-based imaging analysis can examine the composition of different preparation of influenza VLPs to reveal the presence of undesired contaminants.


1:00 Session Break

VECTOR ANALYTICS AND CHARACTERIZATION (CONT.)1:45 Chairperson’s RemarksChristine Le Bec, PhD, Head, Analytical Development, Genethon

1:50 Comparison of in vitro and in vivo Potency Assays to Support Phase I Clinical TrialsLyndi Rice, PhD, Director, Analytics, Gene Therapy Program, University of PennsylvaniaReliable and functional potency assays are key to supporting successful drug product release, stability, and comparability studies. While in vivo potency assays support drug efficacy, many models are difficult to breed. Functional in vitro potency assays offer the benefit of shorter assay length, decrease dependency on colonies, and demonstrate drug potency and transgene function. An example will be discussed regarding two potency assays developed to support clinical trials.

2:20 Development and Qualification of qPCR and dPCR Vector Genome Titering Methods in an Effort to Evolve Gene Therapy Analytical ParadigmsJarrod Dean, Scientist, Analytical Development, Biopharmaceutics Development, Sanofi



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Health authorities emphasize the importance of employing suitable dose-determining methods for gene therapy products. We will discuss the qualification of qPCR and dPCR vector genome concentration platforms in support of gene therapy projects. This presentation spans method development through qualification, highlighting the suitability of dPCR as a platform for titering gene therapy products, and illustrating the benefits of comparing platforms early in product development.

2:50 Analytical Methods for Characterizing Viral VectorsRussell Goetze, PhD, Scientist, Vector Analytics, bluebird bio

3:20 Multiplex RT-QPCR-Based Expression Assay for RAAV-Delivered Gene TherapyDong Xu, PhD, Senior Scientist, Analytical Development, Biogen, Inc.A multiplex reverse transcription quantitative PCR assay was developed to evaluate the expression of a therapeutic transgene delivered by recombinant AAV. It uses a housekeeping gene in human host cells as internal control. The final readout of the assay has been shown to reflect the viral dose at the infection step in a DNA-independent manner.




7:30 End of Day

THURSDAY, AUGUST 16


VECTOR ANALYTICS AND CHARACTERIZATION (CONT.)8:25 Chairperson’s RemarksXiaohui Lu, PhD, Senior Scientist, Analytical Development, Biogen

8:30 Analytical Strategies on Quantification of AAV Empty Capsids to Support Process DevelopmentWei-Chiang Chen, PhD, Scientist I, Bioassay and Gene Therapy, Analytical Development, BiogenOne of the major challenges throughout the process development is to well-characterize empty capsids and remove them effectively. In this presentation, varieties of analytical methods are applied on the same set of samples and compared with their specifications. In our finding, anionic exchange (AEX)-HPLC demonstrates very good correlation with transmission electron microscopy (TEM), and the results of AEX-HPLC are also close to analytical ultracentrifugation (AUC). Finally, strengths and weakness of different methods are summarized and discussed in this presentation.

9:00 Analytical Issues for AAV Gene Therapy ProductsChristine Le Bec, PhD, Head, Analytical Development, GenethonSelected analytical assays were developed to assess the vector productivity, vector purity, biological activity/potency. The quantitative PCR (qPCR) is the current gold method of titrating AAV genomes. The Droplet Digital PCR (ddPCR), a new technology which has been designed for accurate DNA quantification, could improve titer determination. Comparison between these two methods will be discussed. The presentation will also cover methods to determine the ratio of full/empty viral capsids.

Sponsored by9:30 Steps to Validate an in vitro Relative Potency Assay for Gene Therapy Products Karen Doucette, MBA, Director, Operations, ACF BioservicesAlbert J. Owen, PhD, Vice President, Scientific Affairs, ACF BioservicesA validated potency assay is required for approval and lot release of all gene therapy products. The assay must represent the product’s MOA and measure performance relative to a reference standard in a reproducible, controlled manner. This presentation explores advancing an in vitro relative potency assay from development through validation.


VECTOR ANALYTICS AND CHARACTERIZATION (CONT.)10:45 Leveraging ddPCR-Based Assays for AAV Genome Quantitation, Identity, and IntegrityPete Clarner, Senior Associate Scientist, Analytical Development, BiogenddPCR has become a powerful tool for measuring viral vector genome titer and is rapidly replacing qPCR as the assay of choice. We have developed a platform ddPCR method for titering viral vector samples, that also has utility beyond just quantitation. Our assay also provides a genome identity assessment by primarily targeting the gene-of-interest, with additional PCR targets within the vector genome for quality and integrity assessments as well.

11:15 Development of Quantitation Assays for Gene TherapyHema Rao, PhD, Scientist, Sanofi Gene Therapy Skill Center

11:45 Quality Control of Gene TherapiesDavid Malliaros, PhD, Senior Director, QC, Ultragenyx




2ND ANNUAL

Gene Therapy CMC and AnalyticsRegulation, Characterization and Control of Gene Therapies

Gene Therapy



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3RD ANNUAL

Gene Therapy ManufacturingCommercializing Gene Therapies

Gene Therapy

THURSDAY, AUGUST 16




GENE THERAPY PROCESS DEVELOPMENT AND SCALE-UP1:55 Chairperson’s RemarksJohn Pieracci, PhD, Director, Purification, Biogen

2:00 KEYNOTE PRESENTATION: Challenges and Strategies for the Development of a Robust, Scalable, Cost-Effective Biomanufacturing ProcessSadettin Ozturk, PhD, Senior Vice President, Process and Analytical Development, MassBiologicsThe use of viral vectors has increased in recent years, both as gene therapies and as vectors for ex vivo cell therapy products. Industrialization of viral vector manufacturing is maturing as companies tackle problems in process control, scale-up, facility design, characterization and quality, and regulatory considerations. This presentation will examine the current state of the art, emerging technologies and challenges.

2:45 Enabling Industrial Scale Production of Lentiviral Vectors for Gene TherapyKelly Kral, PhD, Associate Director, Vector Process Development and Manufacturing, bluebird bioLentiviral vectors are an ideal platform for indications requiring long-term, stable expression, but the production processes have historically been limited by scale. As the field has now entered commercialization, there is demand for larger quantities of vector, driving the need for more scalable processes. This presentation will review the development, scale-up, and tech transfer of our suspension-based lentiviral vector process.

3:15 Strategies to Deliver Scalable and Reliable Lentiviral Vector BiomanufacturingJeffrey Bartlett, PhD, CSO, Calimmune, Inc.Large-scale clinical production of lentiviral vectors (LV) using current good manufacturing practice (cGMP) methods comes with significant challenges. We have established the Cytegrity stable cell line system for LV bioproduction and have defined key process, quality and regulatory parameters needed to achieve desired productivity and quality across multiples scales and different bioproduction systems. This approach has allowed the production of LV required for Phase I and II clinical trials, while paving the way for future commercialization.

Sponsored by3:45 Evolving Process-Centric Facility DesignMike Sheehan, MSc, MBA, PMP, Senior Project Manager, DPS GroupIncreasingly gene therapy products transitioning from clinical phase to commercial manufacture is driving demand for companies to provide additional capacity. Bringing products to market requires exploring opportunities for leading edge facility design, implementing new & evolving technologies, responding to scalability, speed to market and financial considerations.


4:15 Scalable Lentiviral Vector Production Using HEK293 Suspension CellsParminder S Chahal, Research Officer, Human Health Therapeutics Research Centre, National Research Council CanadaWe have developed expertise in the production of lentiviral vectors (LV) using packaging cell lines and stable producers. Both grow in suspension and in serum-free conditions. Using a stable producer cell line that produces LV expressing GFP, we have compared different modes of operation in bench-scale bioreactors (batch, fed-batch and perfusion). Next, a battery of filters and supplements were evaluated for clarification. A maximal recovery of 78% was obtained.

4:45 Development and Characterization of Novel Micro-RNA Attenuated Oncolytic Herpes Simplex VirusesMichael Paglia, MSc, Vice President, CMC Operations, OncorusOncorus is developing next generation HSV-based oncolytic virus with enhanced potency for tumor cell killing and recruitment of the immune system. Our innovative miR-attenuation strategy enables robust viral replication in tumor cells, while preventing replication in healthy tissue. The development and characterization of therapeutic oHSV requires thorough product understanding gained through process characterization. Strategies for development and characterization of manufacturing processes centered around a strong organizational infrastructure will be presented.

5:15 End of Day

FRIDAY, AUGUST 17


AAV PROCESS DEVELOPMENT8:25 Chairperson’s RemarksNathalie Clément, PhD, Associate Director and Associate Professor, Powell Gene Therapy Center, Pediatrics, University of Florida

8:30 FEATURED PRESENTATION: rAAV Vector Design, Capsid Directed Evolution and Scale Up Activities Using the BEVS SystemJacek Lubelski, PhD., VP, Global Pharmaceutical Development, uniQure



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9:00 Towards a Pivotal Process for AAV Manufacture with HSVDavid Knop, PhD, Executive Director, Process Development, AGTC 9:30 Large-Scale Manufacturing of Clinical Grade AAV in the Academic SettingNathalie Clément, PhD, Associate Director and Associate Professor, Powell Gene Therapy Center, Pediatrics, University of FloridaThe talk will present our current methods for the production of research and clinical-grade rAAV with a special emphasis on the HSV-based suspension method capable of generating high titers of improved rAAV quality. Up-to-date in vitro, in vivo, and clinical data will be shown, and pros and cons of the method will be discussed in comparison to the two other most common methods, transfection and the baculovirus system.


AAV PROCESS DEVELOPMENT (CONT.)10:30 Scale-Up Approach to AAV ManufacturingJohannes C.M. van der Loo, PhD, Director, Clinical Vector Core, The Raymond G. Perelman Center for Molecular and Cellular Therapies, Children’s Hospital of PhiladelphiaThe Clinical Vector Core at the Children’s Hospital of Philadelphia manufactures preclinical- and clinical-grade AAV for academia and industry-sponsored clinical trials. With the field of gene therapy maturing, there is a growing need for larger scale products. We will discuss a strategy for scale-up that builds on our existing mammalian adherent cell-based manufacturing platform.

11:00 Virus-Like Particles and Other Extracellular Particles from Insect and Mammalian CellsAlois Jungbauer, PhD, Professor, Institute of Biotechnology, University of Natural Resources and Life Sciences (BOKU)Virus-like particles and other extra cellular particles are a next generation of biopharmaceuticals. They can be produced by a wide variety of host cells. The challenge is the production of high titers and downstream processing. The particle of interest are contaminated with other particles with similar biophysical properties and therefore difficult to separate. Examples will be given for 3 different cell types.

Sponsored by11:30 Considerations for the Purification Process Characterization of an AAV from Recovery to Drug Substance Ratish Krishnan, PhD, Scientist, Bioprocessing Research & Development, PfizerSmart and efficient approaches for lab-scale characterization are required to ensure a robust adeno-associated manufacturing process. Specific challenges related to the uniqueness of characterizing an AAV manufacturing process will be discussed. Focus will be given to working with limited quantities of material and employing assays that are still being defined.

Sponsored by12:00 pm Next Generation AAV Viral Vector Manufacturing: Proven Technologies with a Modern TwistSandhya Buchanan, Director, Upstream Process Development, FUJIFILM Diosynth BiotechnologiesCurrent approaches to commercial-scale manufacture of viral vectors have been successful for many early phase trials and some late phase trials. Unique challenges/limitations arising for AAV manufacturing include quantities sufficient for patient needs and consumables for manufacturing. We discuss proven technologies blended with modern advancements to meet the needs of the advancing field of gene therapy.


1:15 Session Break

MANUFACTURING GENE THERAPIES1:25 Chairperson’s RemarksChia Chu, Senior Principal Scientist, Bioprocess Research & Development, Pfizer

1:30 FEATURED PRESENTATION: Separation of Full and Empty AAV Particles Using Scalable Isocratic Elution ChromatographyMeisam Bakhshayeshi, PhD, Head, Purification Development, Gene Therapy, BiogenRobust and efficient removal of AAV empty particles is a critical part of the AAV manufacturing process. In this study, we present a scalable ion exchange chromatography process with isocratic wash and elution to separate full and empty particles. A combination of mono- and di-valent salts were used as eluents to achieve the high degree of resolution required for this separation. High product purity and recovery was achieved from this process.

2:00 Lyophilisation of AAV Gene Therapy ProductTanvir Tabish, PhD, Head, Drug Product Development for Gene Therapy, Device and Combination Products, ShireThe gene therapy adeno-associated virus (AAV) subtype 8 containing Factor IX (FIX)(BAX335) was formulated in a new proprietary buffer and lyophilized. A stability study was established with the lyophilized material to determine its stability profile at the accelerated temperature of +5°C over a 10 month period. The freeze-dried product displayed an improved stability profile when stored at a temperature of +5°C. We demonstrated the feasibility of lyophilisation of the AAV viral drug product in the formulation buffer.

2:30 AAV Manufacturing at 2,000L ScaleAlex Fotopoulos, PhD., Senior Vice President, Technical Operations, Ultragenyx

3:00 CMO Selection for Cell & Gene TherapyChad Green, PhD, Principal & Senior Consultant, Dark HorseAs the diversity of CMOs available for cell and gene therapies continues to grow

3RD ANNUAL


Gene Therapy



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3RD ANNUAL


Gene Therapy

worldwide, identifying the most suitable to engage is becoming an increasingly complex challenge. This presentation will address fundamental questions, such as whether a CMO is even the best choice for manufacturing before progressing to provide concrete guidance on the critical questions to ask prospective CMOs (and yourself), how to ask them and how to analyze the answers and make an optimal, rational choice.




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Manufacturing The theme of the Manufacturing stream this year is Re-imagining the Future of Manufacturing. Bringing together emerging concepts and proven strategies, the conferences aim to challenge the industry to re-think manufacturing, from re-defining the processes to re-designing the facilities, and introducing AI, machine learning and big data into process modeling and analytics. Join us as we explore the new frontiers in biopharmaceutical manufacturing.

AUGUST 13-14

AGENDA Continuous Processing in Biopharmaceutical Manufacturing

AUGUST 15-16

AGENDA Manufacturing 4.0

2018 MANUFACTURING TRACKS



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INAUGURAL

Manufacturing 4.0Where A.I. Meets Bioprocessing

Manufacturing

FOR CONTINUOUS PROCESSING IN BIOPHARMACEUTICAL MANUFACTURING

AGENDA taking place Monday, August 13 and Tuesday, August 14, please click here.



DISRUPTIVE TECHNOLOGIES – ENABLING PERSONALIZED MEDICINE, HEALTHCARE AND RAPID RESPONSE8:05 Chairperson’s RemarksRobert Dream, PE, CPIP, Managing Director, HDR Company

8:15 KEYNOTE PRESENTATION: Disruptive Innovations to Lower Healthcare CostsGovind Rao, PhD, Professor and Director, Center for Advanced Sensor Technology, UMBCRising healthcare costs are a major threat to the economy. While much of the focus has been on healthcare access, not much attention has been paid to reducing healthcare costs. We demonstrate novel technological approaches that promise to harness the revolutions demonstrated by the consumer electronics and computer industry that show that quality and cost are not synonymous. We demonstrate disruptive low-cost diagnostic and therapeutic technologies that can empower caregivers with novel, low-cost point-of-care solutions.

9:00 Cell Free Synthesis: A Highly Disruptive Technology for Manufacture of Personalized/Targeted MedicinesDaniel G. Bracewell, PhD, CEng, MIChemE, Professor, Bioprocess Analysis & Doctoral Admissions Tutor, Department of Biochemical Engineering, University College LondonThe pipeline of personalized and targeted medicines covers a wide range of treatment types including cell and gene therapies, vaccines based on peptides or proteins, and protein-based therapies. These increasingly diverse and smaller market size products require improved agility and productivity in process design if manufacture is to be affordable. We examine the potential of cell free synthesis, to address this need, focusing on protein-based therapeutic products.

9:30 Precision Health and 3D Bioprinting: A Head to Toe WorkflowPaul C. Goodwin, MSc, Scientific Director, GE HealthcareCurrent bioprocess manufacturing focuses on biologic replacement therapies and monoclonal antibodies. We are at the beginning of a new wave of therapies wherein the cell is the therapy. We have seen investments and advancements in T-cell therapies, but if they only address immunotherapy, we will have missed a larger opportunity. We present here the vision of a workflow to address the need to generate replacement structures, tissues, and organs that are living, bespoke, autologous, and regulated.


10:45 Accelerating Development of Medical Countermeasures for the Warfighter: Platform Technologies as Critical Tools to Reduce Developmental Risk and Costs, and Enable Rapid Responsiveness to Novel ThreatsChristopher Earnhart, PhD, Joint Product Lead, PRISM; Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense, Department of DefenseThe DoD PRISM (Platforms for Rapid Integrated Solutions for Medical Countermeasures) office is investigating mechanisms to accelerate medical countermeasure (MCM) development and fielding, primarily by using platform technologies. PRISM is adapting platforms to meet Warfighter requirements and provide MCMs against known threats at reduced cost, schedule, and developmental risk. Additionally, PRISM is implementing innovative processes, technologies, and regulatory interactions to enable response within an operationally relevant timeframe to emerging threats.

11:15 Streamlining the Cell Therapy Workflow with Silicon Chip TechnologySunil Gangadharan, PhD, Strategic Partnerships, Life Science Technologies and Smart Electronics Applications, imec A major hurdle to scale up the market of cell therapies is the cost and complexity of the workflow. We propose a novel approach based on a standardised workflow which will be easy to use, reliable and reproducible. Using silicon chip technology, fully integrated solutions can be built, combining microfluidics, photonics-based biosensors, PCR, and single cell manipulation. We will present a portfolio of technologies which will be the building blocks for bioprocessing and delivery of future personalised therapies.

11:45 Digital Manufacturing Use Cases from Different Industries: Applicability to BioPharmaNamit Mehta, PhD, Director, Health Industries - Operations Strategy, Strategy& (Part of the PwC Network)Digital manufacturing is a solution geared towards solving traditional supply chain challenges through application of cutting edge technologies like Big data, 3D printing, IoT, AI etc. These platforms are creating positive impacts measurable in both the top line & bottom line of companies. We are looking at a few success stories from different industries where application of such technologies have given remarkable results. We are also gauging the application of the same towards the BioPharma industry where they are geared to solve problems across the value chain.


1:00 Session Break

INDUSTRY 4.01:45 Chairperson’s RemarksMichael Sokolov, PhD, Postdoctoral Fellow and Lecturer, Institute of Chemical and Bioengineering, ETH Zurich and Co-Founder & COO, DataHow



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Manufacturing



1:50 Industrie 4.0: What’s NextRobert Dream, PE, CPIP, Managing Director, HDR CompanyIndustry 4.0 can solve key challenges facing manufacturers – from extreme supply, demand, and design variability, to emerging markets of one, to the growing need for rapid innovation. Leveraging the principles of Industry 4.0 – and its enabling technologies to automate, integrate, and optimize manufacturing processes – companies can shorten cycle times, improve product quality, and implement efficiency across their operations, as well as grow the manufacturing of highly customized products on a global scale.

2:20 Industry 4.0 and Continuous Biopharmaceutical ManufacturingSamet Yildirim, MSc, MBS, Manager, Technology & Innovation, Global Technology Management, Boehringer Ingelheim Fremont, Inc.This talk will address why integrated continuous manufacturing is essential to move into the Industry 4.0 and how we can utilize digital technologies in biopharma manufacturing. Questions explored include: What industrial revolution are we currently in? Why disposable, integrated and continuous process is essential for industry 4.0? Highly automated and modular flexible facilities are the future of biomanufacturing. Knowledge management, machine learning and big data are essential parts of the game. How can 3D printing be part of disposable processes?

2:50 Breakout Discussions

• Big Data in Bioprocessing – Vice or Virtue? Moderator: Michael Sokolov, PhD, Postdoctoral Fellow and Lecturer, Institute of

Chemical and Bioengineering, ETH Zurich and Co-Founder & COO, DataHow

• The Path of Least Resistance, and the Adoption of New Technology Moderator: Eben Crawford, Process Engineer, Celldex Therapeutics




7:30 End of Day

THURSDAY, AUGUST 16


MACHINE LEARNING, DATA ANALYTICS AND MODELING8:25 Chairperson’s RemarksDaniel G. Bracewell, PhD, CEng, MIChemE, Professor, Bioprocess Analysis & Doctoral Admissions Tutor, Department of Biochemical Engineering, University College London

8:30 Mastering the Digital Challenge in Bioprocessing through Machine Learning and Hybrid ModelingMichael Sokolov, PhD, Postdoctoral Fellow and Lecturer, Institute of Chemical and Bioengineering, ETH Zurich and Co-Founder & COO, DataHowFollowing the advanced examples of process digitalization and automation in industries such as automotive, several important trends could be observed in the biopharmaceutical domain. In particular, continuous bioprocessing and continuous data acquisition as well as the utilization of data- and knowledge-driven tools for process analysis and control thrive towards the standards and goals of industry 4.0. Based on industrial case studies this presentation will highlight the important roles of advanced process modeling for bioprocess digitalization.

9:00 Model-Based Tools for Automated Bioprocess DevelopmentMariano Nicolas Cruz Bournazou, PhD, Research Associate, Bioprocess Engineering, Technische Universität BerlinThe internet of things in biotechnology will be always coupled with experimental activities to validate and re-adjust models to the reality of living systems. Thus robotic laboratories have to be intelligent when designing experiments and learning from the data. We present an integrated intelligent laboratory for rapid phenotyping of biosystems including: easy interaction of all devices, experimental design

9:30 Integrated E2E Workflow System for Data- and Knowledge-Driven Process DevelopmentAmanda Fitzgerald, PhD, Senior Scientific Consultant, Biologics, GenedataWe present an E2E system for the development of manufacturing processes for biotherapeutics supporting workflows from post-discovery to commercial manufacturing. The system integrates the development and assessment of cell lines, upstream and downstream processes, corresponding raw materials, and analytical tests. We show how the system tracks the full history of all manufacturing cell lines, as well as USP and DSP batches, and enables automation via integration with liquid handling systems, bioreactors, and other instruments.


10:45 Data Mining Benchtop Experiments for Realtime Manufacturing Analysis and Facility DevelopmentEben Crawford, Process Development Engineer, Celldex Therapeutics



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INAUGURAL


Manufacturing



11:15 CLOSING PANEL DISCUSSION: Advance Technologies and its Impact on ManufacturingRobert Dream, Managing Director, HDR CompanyIndustrie 4.0 involves the complete automation and computerization of the manufacturing processes, utilizing the collective potential of computerization and automation to revolutionize how the manufacturing industry works. The major technologies that would be contributing to Industrie 4.0 include IoT (Internet of Things), Cyber-physical systems, Cloud Computing all together creating “Smart factory”. Advancement of technologies and utilizing Industrie 4.0 will impact;• Big data• Improved production efficiency• Smart organizations• Improved quality of products and services• Better control of the industrial processes• Sufficient data for better decision making• Easier standardization






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SPONSORSHIP & EXHIBITOR OPPORTUNITIES

REGISTER AS AN EXHIBITOR

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For more information regarding exhibit and sponsorship,

please contact:

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Sherry Johnson Senior Manager, Business Development

781-972-1359 [email protected]

COMPANIES L-Z:

Carolyn BentonManager, Business Development

781-972-5412 [email protected]

Comprehensive sponsorship packages allow you to achieve your objectives before, during, and long after the event. Signing on earlier will allow you to maximize exposure to hard-to-reach decision-makers.

PODIUM PRESENTATIONS — Available within Main Agenda!Showcase your solutions to a guaranteed, targeted audience through a 15- or 30-minute presentation during a specific conference program, breakfast, lunch, or separate from the main agenda within a pre-conference workshop. Package includes exhibit space, on-site branding, and access to cooperative marketing efforts by CHI. For the luncheon option, lunches are delivered to attendees who are already seated in the main session room. Presentations will sell out quickly, so sign on early to secure your talk!

ONE-ON-ONE MEETINGSSelect your top prospects from the pre-conference registration list. CHI will reach out to your prospects and arrange the meeting for you. A minimum number of meetings will be guaranteed, depending on your marketing objectives and needs. A very limited number of these packages will be sold.

INVITATION-ONLY VIP DINNER/HOSPITALITY SUITESponsors will select their top prospects from the conference pre-registration list for an evening of networking at the hotel or at a choice local venue. CHI will extend invitations and deliver prospects, helping you to make the most out of this invaluable opportunity. Evening will be customized according to sponsor’s objectives. (i.e.: Purely social, Focus group, Reception style, Plated dinner with specific conversation focus).

EXHIBITExhibitors will enjoy facilitated networking opportunities with 1,200+ qualified delegates, making it the perfect platform to launch a new product, collect feedback, and generate new leads from around the world. Exhibit space sells out quickly, so reserve yours today!

Additional branding & promotional opportunities include:• Hotel Room Keys• Footprint Trails• Conference Tote Bags• Badge Lanyards SOLD!• Literature Distribution (Tote Bag Insert or Chair Drop)• Padfolios• Program Guide Advertisement

Looking for additional ways to drive leads to your sales team?CHI’s Lead Generation Programs will help you obtain more targeted, quality leads throughout the year. We will mine our database of 800,000+ life science professionals to your specific needs. We guarantee a minimum of 100 leads per program!Opportunities include:• Live Webinars• White Papers• Market Surveys• Podcasts and More!



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HOTEL & TRAVEL INFORMATION

Top Reasons to Stay at the Sheraton Boston• No Commute - conference takes place at the hotel• Complimentary Wireless Internet in your guest room• Take advantage of the deeply discounted $265 group rate – an amazing

rate for this area and time of year!!!• Hundreds of restaurants and shops are just steps from the hotel• Local attractions including Fenway Park, Museum of Fine Arts and

Boston Public Gardens within walking distance from hotel

Conference Venue and Hotel:Sheraton Boston 39 Dalton Street Boston, MA 02199 Phone: 617-236-2000

Discounted Room Rate: $265 s/d Discounted Cut-off Date: July 17, 2018



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Reservations: Please visit the Travel page of BioprocessingSummit.com for further details and to book your hotel.





PRESENT A POSTER & SAVE

$50Cambridge Healthtech Institute encourages attendees to gain further exposure by presenting their work in the poster sessions. To secure a poster board and inclusion in the conference materials, your abstract must be submitted, approved and your registration paid in full by July 13, 2018.

n Your research will be seen by leaders from top pharmaceutical, biotech, academic and government institutesn Your poster abstract will be published in the conference materialsn Receive $50 off your registration fee

LEARN MORE



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http://www.bioprocessingsummit.com/bpd_content.aspx?id=89772




CONFERENCE DISCOUNTSPoster Submission - Discount ($50 Off): Poster abstracts are due by July 13, 2018. Once your registration has been fully processed, we will send an email containing a unique link allowing you to submit your poster abstract. If you do not receive your link within 5 business days, please contact [email protected]. *CHI reserves the right to publish your poster title and abstract in various marketing materials and products.REGISTER 3 - 4th IS FREE: Individuals must register for the same conference or conference combination and submit completed registration form together for discount to apply.Alumni Discount: Cambridge Healthtech Institute (CHI) appreciates your past participation at The Bioprocessing Summit. As a result of the great loyalty you have shown us, we are pleased to extend to you the exclusive opportunity to save an additional 20% off the registration rate.Group Discounts: Discounts are available for multiple attendees from the same organization. For more information on group rates contact Uma Patel, [email protected], 781-972-5447.

If you are unable to attend but would like to purchase The Bioprocessing Summit CD for $750 (plus shipping), please visit BioprocessingSummit.com. Massachusetts delivery will include sales tax.

ADDITIONAL REGISTRATION DETAILSEach registration includes all conference sessions, posters and exhibits, food functions, and access to the conference proceedings link.Handicapped Equal Access: In accordance with the ADA, Cambridge Healthtech Institute is pleased to arrange special accommodations for attendees with special needs. All requests for such assistance must be submitted in writing to CHI at least 30 days prior to the start of the meeting.To view our Substitutions/Cancellations Policy, go to www.healthtech.com/regdetailsVideo and or audio recording of any kind is prohibited onsite at all CHI events.

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CONFERENCE & TRAINING SEMINAR PRICING

PREMIUM PACKAGE - BEST VALUE! (Includes access to all conferences and training seminars Monday-Friday. Excludes short courses.)

Registrations after July 20, 2018 and on-site $3295 $1695

STANDARD PACKAGE (Includes access to two conferences and/or training seminars. Excludes short courses.)


BASIC PACKAGE (Includes access to one conference or training seminar. Excludes short courses.)


SHORT COURSE PRICING(Includes access to short courses only)

Full-Day Workshop (Life Science Venture Valuation) $895 $595

One short course $595 $345

Two short courses $895 $595

Three short courses $1095 $695

How to Register: [email protected] • P: 781.972.5400 or Toll-free in the U.S. 888.999.6288Please use keycode 1881F when registering

Practical Solutions for Today’s Bioprocess ChallengesAUGUST 13-17, 2018 | SHERATON BOSTON | BOSTON, MA

TENTH ANNUAL



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Practical Solutions for Today’s Bioprocess Challenges