RETURNING IN-PERSON. RETURNING SAFELY

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FEATURED PLENARY & KEYNOTE SPEAKERS

Bioproduction David Maraldo, PhD Merck & Co., Inc.

Lessons Learned from the Pandemic Darrin Cowley, PhD AstraZeneca

Upstream ProcessingWolfgang Paul, PhD UCB

Downstream Processing Sonja Berensmeier, PhD Technical University of Munich

Analytical & QualityKrishnan Sampathkumar, PhD MacroGenics, Inc.

Stability & ImpuritiesChristopher J. Roberts, PhD University of Delaware

Vaccine ManufacturingUlrich Blaschke, PhD BioNTech

Gene Therapy Mark Galbraith, PhD Spark Therapeutics

Cell TherapyTam Soden, PhD Kite Pharma

mRNA Vaccines: A Paradigm Shift Sudha Chivukula, PhD Sanofi Pasteur Biologics Co.

Manufacturing Next-Generation TherapiesMartha Rook, PhD Sigilon Therapeutics, Inc.

Sustainability and the Future of BioprocessingKristi Budzinski, PhD Genentech, Inc.

August 16-19, 2021

Attend In-Person or Virtually

2 Ways to Attend, 1 Shared Experience

Final Days to Register! Reserve Your Spot Now!

CambridgeHealthtech InstituteOrganized by

Premier Sponsors

Sheraton Boston & Online

RETURNING IN-PERSON.RETURNING SAFELY.

SOLVING TODAY’S CHALLENGES,LEADING TO TOMORROW’S ADVANCES

http://www.BioprocessingSummit.com

Table of Contents

Event at-a-Glance

Plenary Keynote Presentations

Sponsorship Programs

Venue Information

Pricing & Registration

NEW Flexible Registration

For more than 10 years, The Bioprocessing Summit has been recognized as the premier forum for industry leaders to share the latest research in bioprocess R&D, scale-up, quality and analytics. As we navigate the new realities of conferences and gatherings you can count on CHI’s commitment to deliver quality content and facilitated networking opportunities to help achieve your research goals.

2 Ways to Attend, 1 Shared ExperienceYour safety and comfort are our priority. To provide maximum flexibility to match your needs, CHI will provide this event live in-person and virtually. We feel this combination will bring about one truly unique event experience. This new conference norm will fuse the best of our traditional onsite events with the expanded benefits of a virtual conference. Bringing together the life sciences community is what CHI does, and we look forward to bringing our community together with this new offering.

Experience the Most Immersive Learning & Networking Event for the Bioprocessing Industry

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CHI’s Mandatory COVID-19 Vaccination Policy Your Safety is Our Top Priority

To ensure maximum safety, CHI has instituted a mandatory COVID-19 vaccination policy for all in-person participants of our events. Attendees will be asked to furnish proof of vaccination. Additional details on the vaccine policy will be provided upon registration.Attendees that cannot participate because of this policy, or due to travel restrictions, are encouraged to participate using our virtual event platform. CHI virtual events provide you with an in-person experience at your convenience, anywhere, anytime.

Our Code of ConductAll in-person attendees must agree to CHI’s Code of Conduct

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IN-PERSON BENEFITS

Seamlessly switch between In-person and/or virtual registrationSelect an In-person or virtual option, and you have the flexibility to switch your preferred event experience at any time leading up to the conference. Simply contact us, and we will either charge you the difference for upgrading to in-person or credit back the price for transferring to virtual. Our flexible registration is designed to take the uncertainties out of these uncertain times.

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Recapture the power of serendipitous face-to-face connections with colleagues and technology providers who share similar interests and challenges

Connect and schedule 1:1 meetings with the right onsite and virtual attendees based on CII’s matching algorithm that aligns areas of mutual interest and expertise

Join most speakers live and in-person while watching others On-Demand at your convenience

Take advantage of our flexible registration policy that allows you to easily transfer at any time between in-person and virtual participation

Connect in-person with sponsors and exhibitors under responsible safety and social distancing protocols

Access in-person and virtual exhibit booths, posters, and networking interactive discussions

PLUS all of the virtual benefits below






CURRENT SPONSORS

Premier Sponsors

Corporate Sponsors

Corporate Support Sponsors

(as of 7/16/21)






Cell Culture & Cell Line Optimization

Stream #1 UPSTREAM PROCESSING

Continuous Processing in Biopharm Manufacturing

Stream #2 DOWNSTREAM PROCESSING

Host Cell ProteinsStream #3 ANALYTICAL & QUALITY

Rapid Methods to Assess Quality & Stability of Biologics

Stream #4 STABILITY & IMPURITIES

Gene Therapy CMC and AnalyticsStream #5 GENE THERAPY

Gene Therapy CMC and Analytics Stream #6 CELL THERAPY

Vaccine Development and Manufacturing

Stream #7 VACCINE MANUFACTURING

Bioproduction: Scale, Bioreactors & Digitalization

Advances in Purification & Recovery

Accelerating Analytical Development

Detection, Characterization and Control of Impurities in Biologics

Gene Therapy Manufacturing

Cell Therapy CMC and Analytics

Bioproduction: Scale, Bioreactors & Digitalization

CONFERENCE PROGRAMS AUGUST 16-17 AUGUST 18-19

Cambridge Healthtech Institute encourages attendees to gain further exposure by presenting their work in the poster sessions. To secure an onsite poster board and/or ensure your virtual poster presentation is included in the conference materials, your full submission must be received, and your registration paid in full by July 9, 2021.Register and indicate that you would like to present a poster. Once your registration has been fully processed, we will send an email with a unique link and instructions for submitting your abstract and other materials. Please see website for more information.

PRESENT A POSTER & SAVE $50*! Live In-Person or

Virtual Real-Time

Reasons you should present your research poster at this conference:• Your research will be seen by our

international delegation, representing leaders from top pharmaceutical, biotech, academic and government institutions

• Discuss your research and collaborate with other attendees

• Your poster presentation will be published in our conference materials

• Receive $50 off your registration*

LEARN MORE »

EVENT AT-A-GLANCE

*This discount does not apply to product or service providers.


https://www.bioprocessingsummit.com/posters





PLENARY KEYNOTE SESSIONS August 16 & 18

All Times EDT

LEADING TO TOMORROW’S ADVANCESWEDNESDAY, AUGUST 18

3:50 pm Plenary Keynote IntroductionDominic Clarke, PhD, ISCT Process & Product Committee Co-Chair & Chief Technology Officer, Cell and Gene Therapy, Discovery Life Sciences

4:00 Manufacturing Next-Generation TherapiesMartha Rook, PhD, Chief Technical Operations Officer, Sigilon Therapeutics, Inc.Cell and gene therapies have shown dramatic clinical progress in recent years. Driven by this clinical success and the needs of commercialization, manufacturing strategies are progressing. Nevertheless, a standard manufacturing template for these therapies has not evolved and with novel modalities continuing to emerge we may be in a post-template manufacturing landscape. CMC strategies must be developed to avoid manufacturing becoming a roadblock to therapeutic success.

4:30 Sustainability and the Future of BioprocessingKristi Budzinski, PhD, Principal Product Stewardship & Green BioPharma Manager, Genentech, Inc.The future of bioprocessing demands flexible, scalable solutions that can accommodate the rapidly evolving landscape of biopharmaceutical products while also minimizing impact on the environment. This talk will highlight some of the major opportunities for reducing the environmental impact of bioprocessing through the application of metrics such as process mass intensity and lifecycle assessment methodology. Results will be presented from both a Genentech perspective and industry-wide perspective.

SOLVING TODAY’S CHALLENGESMONDAY, AUGUST 16

4:20 pm Plenary Keynote IntroductionJames Warren, PhD, Vice President, Pharmaceutical Development, Ultragenyx Pharmaceutical

4:30 mRNA Vaccines: A Paradigm Shift in Pandemic PreparednessSudha Chivukula, PhD, Head, mRNA Technology, Sanofi Pasteur Biologics Co.The rapid development for clinical proof-of-concept and bioprocess scale-up leading to commercial manufacturing and approval under emergency use authorization of COVID mRNA vaccines highlights the potential for an mRNA platform to address future pandemics as well as other unmet public health needs. The framework for optimizing novel mRNA vaccines and formulations, which could include adaptation to monovalent and multivalent vaccines, delivery and balanced immune responses to address emerging viral pathogens such as SARS-COV-2 and pandemic Influenza, will be discussed.

5:00 Lessons Learned from the PandemicDarrin Cowley, PhD, Vice President & Head, Developmental Quality Biologics, Quality Lead COVID Vaccine, AstraZenecaDuring the pandemic, there had to be focus in several areas. Primarily, the safety of the workforce and allowing front line operators to function unhindered. Management needed to change its ways of working, prioritize and create the environment for optimal working. Decision-making and digital tools were implemented and an altered culture was created. Ways of dealing with virtual inspections were also developed.






Upstream Upstream ProcessingProcessingThe need for higher titer, higher producing cell lines, improved cell quality, lower production cost and accelerated development etc., are giving rise to new tools and technologies in upstream processing. Trends such as omics, modeling, gene editing, single-use technologies and digitalization are finding their way into biopharmaceutical companies’ process development strategies and toolkits. In addition to featuring these exciting trends, this year’s Upstream Processing will also feature a dedicated session on “Bioprocess Development Under Pandemic Conditions”, to address the challenges of manufacturing to meet global health, and adapting operations under such adverse conditions.

Cell Culture & Cell Line Optimization

Conference Programs

AUGUST 16-17

Bioproduction: Scale, Bioreactors

& DigitalizationAUGUST 18-19






17th Annual

Cell Culture & Cell Line OptimizationImproving Upstream Productivity and Biologics Quality August 16-17

All Times EDT


MONDAY, AUGUST 16

9:00 am Main Conference Registration

ADVANCES IN CELL CULTURE PROCESS DEVELOPMENT – DIGITALIZATION AND MODELING

9:55 Chairperson’s RemarksSusan Sharfstein, PhD, Professor, Nanobioscience, Nanoscale Science and Engineering, SUNY Polytechnic Institute

10:00 KEYNOTE PRESENTATION: Driving Digital Transformation in Cell Culture Process DevelopmentWolfgang Paul, PhD, Head, Upstream Process Science, UCB

Integration of digital transformation within the process development of Cell Culture processes is a chance and a challenge at the same time. The key for success is to show real applications and demonstrate benefits for process development. Less experiments and more efficient data analysis enable acceleration of process development. Examples for next generation cell culture process development will be highlighted during the talk.

10:30 Modeling Framework to Predict Product Quality Attributes of Cell Culture ProcessesThomas Villiger, PhD, Senior Manufacturing Scientist, Biogen International GmbHReal-time monitoring of biopharmaceutical processes is a key necessity for quality by design manufacturing. This talk describes a platform for the integration and modeling of online process data in combination with spectroscopic sensors to predict product quality in real-time. As a proof-of-concept study, the developed model platform was used to predict the time and process dependent evolution of N-linked glycosylation of a monoclonal antibody in small and large scale bioreactors.

11:00 Generic Hybrid Model for CHO Cultivation Processes to Predict Quality Attributes Across Different Biopharmaceutical ProductsLiliana Montano Herrera, Postdoctoral Researcher, Bioprocess Development, Boehringer Ingelheim Pharma GmbH & Co. KGIn collaboration with DataHow AG, we present a generic hybrid modelling approach able to predict process dynamics of CHO cultivation processes and final quality attributes across different products as a function of the process design in the production bioreactor. This novel approach could support development and process characterization activities by capturing common trends in process performance across various projects and thereby improving prediction capabilities and allowing for less experimental effort.

11:30 CO-PRESENTATION: A New Single-Cell Cloning & Imaging Platform and Scale-Down Bioreactors for Generating Stable Producer Cell LinesJason Nowacki, Sales Executive, CYTENA (A CELLINK Company)Speaker II To Be AnnouncedWe’ll introduce CYTENA´s latest technologies:- F.SIGHT 2.0: ultra-fast single-cell dispenser- UP.SIGHT: combines single-cell dispensing with a new imaging technology for assuring monoclonality- S.NEST: highly parallel scale-down bioreactor for early suspension culture in 96-well platesThe combination of these technologies results in a significant reduction of the timeline for generating stable cell lines for the production of complex antibodies and viral vectors, especially in fully automated workstations.

12:00 pm Enjoy Lunch on Your Own

OMICS AND OTHER PLATFORMS FOR CELL LINE SELECTION & CLONE DEVELOPMENT

12:50 Chairperson’s RemarksSusan Sharfstein, PhD, Professor, Nanobioscience, Nanoscale Science and Engineering, SUNY Polytechnic Institute

12:55 Novel Post-Transcriptional Cell Selection Strategies for Rapid Clone Development and Stability ScreeningSusan Sharfstein, PhD, Professor, Nanobioscience, Nanoscale Science and Engineering, SUNY Polytechnic InstituteCurrently, stable CHO cell lines producing therapeutic, recombinant proteins are established by antibiotic and/or metabolic selection. Here I will describe a novel post-transcriptional selection technology that utilizes an siRNA cloned upstream of the gene of interest, which is processed to produce functional siRNAs, enabling cell enrichment. This presentation describes the selection principle and compares the productivity, doubling time and stability of clones with those developed by conventional selection methods.

1:25 The PICRH Genome and Its Use to Identify Hotspots for Targeted IntegrationKelvin H. Lee, PhD, Gore Professor, Chemical & Biomolecular Engineering, University of DelawareThis presentation will discuss the latest reference genome for the community that has chromosome-length scaffold sequences. In addition, we will discuss the use of genome-scale analyses to identify possible safe harbor regions for targeted integration of transgenes.





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1:55 A Platform Approach to Cell Line Development to Enable Intensified Gene to GMP TimelinesFay Saunders, PhD, Head, Mammalian Cell Culture, PD, FUJIFILM Diosynth Biotechnologies• Apollo™X is a cell line development platform, incorporating next

generation screening technology, enhanced media selection and technical expertise.

• Cell line development timeline reductions have been achieved through the introduction of a novel single cell cloning method which generates high producing clonal cell lines with both high probability and assurance of monoclonality.

• Incorporation of Targeted Locus Amplification (TLA) technology allows the rapid genetic characterization of clonal cell lines

2:25 Networking Refreshment Break

2:40 Multi-Omics Characterization for Early Stage Development of Cell Therapy Products and Manufacturing ProcessesCarolyn Yeago, PhD, Associate Director, Marcus Center for Therapeutic Cell Characterization and Manufacturing, Georgia Institute of TechnologyUnderstanding Critical Quality Attributes (CQAs) and Critical Process Parameters (CPPs) of cell therapy products is essential for scalable, reproducible, and cost-effective manufacturing. Unlike small-molecule drugs or biologics, cells are highly complex systems whose properties can change during ex-vivo manipulation. We describe multi-omics approaches to consider during development of potential cell therapies that can be used to better identify and correlate key attributes to performance and ultimately identify process parameter specifications.

3:10 Optimized Cell Culture Medium at Your Fingertips with At-Line Spent Media AnalysisGraziella Piras, PhD, Bioprocessing Segment Marketing Director, Marketing, 908 DevicesCell culture medium optimization is the fine tuning of key nutrients to reach a cell line productivity potential. It is an iterative process of spent medium analysis combined with a DOE approach. Presented here is a new automated benchtop analyzer for rapid cell culture medium analysis and optimization. The data provided by this novel at-line tool may accelerate process development analytics by days to weeks with a simple approach offering more frequent visibility into nutrient levels for various applications.

3:40 Session Break and Transition to Plenary Keynote

PLENARY KEYNOTE SESSION: SOLVING TODAY’S CHALLENGES

4:20 Plenary Keynote IntroductionJames Warren, PhD, Vice President, Pharmaceutical Development, Ultragenyx Pharmaceutical

4:30 KEYNOTE PRESENTATION: mRNA Vaccines: A Paradigm Shift in Pandemic PreparednessSudha Chivukula, PhD, Head, mRNA Technology, Sanofi Pasteur Biologics Co.

The rapid development for clinical proof-of-concept and bioprocess scale-up leading to commercial manufacturing and approval under emergency use authorization of COVID mRNA vaccines highlights the potential for an mRNA platform to address future pandemics. The framework for optimizing novel mRNA vaccines and formulations delivery and balanced immune responses to address emerging viral pathogens such as SARS-COV-2 and pandemic Influenza, will be discussed.

5:00 KEYNOTE PRESENTATION: Operating During a Global Pandemic: Lessons Learned from the PandemicDarrin Cowley, PhD, Vice President & Head, Developmental Quality Biologics, Quality Lead COVID

Vaccine, AstraZenecaDuring the pandemic, there had to be focus in several areas. Primarily, the safety of the workforce and allowing front line operators to function unhindered. Management needed to change its ways of working, prioritize and create the environment for optimal working. Decision-making and digital tools were implemented and an altered culture was created. Ways of dealing with virtual inspections were also developed.

5:30 Welcome Reception in the Exhibit Hall with Poster Viewing

6:30 Close of Day





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TUESDAY, AUGUST 17

7:30 am Registration Open and Morning Coffee

IMPROVING CHO CELL PRODUCTIVITY & QUALITY 7:55 Chairperson’s Opening RemarksCarolyn Yeago, PhD, Associate Director, Marcus Center for Therapeutic Cell Characterization and Manufacturing, Georgia Institute of Technology

8:00 Volume and Agitation Speed Influences the Performance of Recombinant CHO Cells in Spin Tube BioreactorsAlan Dickson, PhD, Professor of Biotechnology; Director, Centre of Excellence in Biopharmaceuticals, Manchester Institute of Biotechnology, The University of Manchester

8:30 Boosting Cell-Specific Antibody Productivities in CHO by MTA (Methylthioadenosine) Addition: Quantitative Subcellular Analysis and Process OptimizationRalf Takors, PhD, Professor & Director, Institute of Biochemical Engineering, University of StuttgartIncreasing cell specific productivities (CSPs) is key to improve performance of biopharmaceutical production processes using mammalian cells. Searching for CSP boosting additives, we observed 57% CSP and 50% titer rise in IgG-1 producing CHO-DP12 cells after the addition of S‐(5′‐adenosyl)‐L‐methionine (SAM). Detailed analytics revealed the degradation product MTA (5′‐deoxy‐5′‐(methylthio)adenosine) as the key inductor molecule. Cellular responses were analyzed in detail regarding cell cycle, cell volume and metabolic flux patterns.

9:00 Early Detection of Viability Loss of Producer Cells in a BioprocessMichael Butler, PhD, Principal Investigator, Cell Technology, National Institute for Bioprocessing Research & Training (NIBRT), IrelandContinuous optical and dielectric methods of monitoring mammalian cells in bioprocesses allow the detection of metabolic and morphological changes well before the loss of cell viability as determined by dye exclusion methods such as trypan blue. The presentation will evaluate these alternative methods that allow real-time analysis of the state of cells during production and the possibility of intervention in the bioprocess before loss of viability by the cell population.

9:30 Scaling Up with Orbital Shaken BioreactorsMichael Keebler, PhD, Product Manager, Applied Technologies, Kuhner Shaker, Inc.Single-use bioreactors are widely used instruments for scaling up bioprocesses. Orbital shaken bioreactors, like the Kuhner SB series, offer many advantages in contrast to alternative technologies like stirred-tank and wave bioreactors. Here we review application data on the relative performance of this technology across scale and highlight some of the key features of the SB series that make it an elegant solution for scaling any mammalian bioprocess.

10:00 Coffee Break in the Exhibit Hall with Poster Viewing

10:45 Optimization of Production Bioreactor Feeding Strategy to Mitigate Amino Acid MisincorporationVictoria Drake, Associate Scientist II, Upstream Process Development, Alexion Pharmaceuticals, Inc.Amino acid sequence variants in protein therapeutics have the potential to affect product safety and efficacy. In this case study, significant levels of amino acid misincorporation were identified for a mAb produced by CHO cells. Amino acid misincorporation levels were correlated with amino acid depletion and unbalanced amino acid ratios. An iterative approach was taken to optimize the production bioreactor feeding strategy to successfully mitigate amino acid misincorporation.

11:15 Generation and Characterization of a Highly Optimized, HeLa 3.0 Producer Cell Line (PCL) Production PlatformMatthew Fuller, PhD, Director, Vector Platform Research, UGT Research, Ultragenyx PharmaceuticalA key challenge facing the gene therapy field today is ensuring that manufacturing capabilities surpass current standards to ensure accessibility and affordability for all eligible patients. We have developed a HeLa-based rAAV manufacturing platform with demonstrated scalable 2000L production to support ongoing Phase I/II clinical trials. Recently, we have further optimized our HeLa Producer Cell Line (PCL) platform to HeLa 3.0 by genetically modifying existing, highly productive monoclonal PCLs.

11:45 Correlation of Online and Offline Techniques to Measure CHO Cell Health for Early Detection and Control of Cell StressKyle P. McHugh, PhD, Principal Scientist, Upstream Biologics Process Development, Bristol-Myers Squibb Co.Advances in automated bioreactor sampling and machine learning for imaged-based cell analysis have significantly improved our ability to quantify and monitor changes in cell health for bioprocessing development. Here we describe leveraging multiple methods of measuring cell health including the Canty cell imager with Flownamics SegFlow autosampler, biocapacitance probes, image-based and flow cytometry, and standard trypan blue-based quantitation to develop more representative models for online or at-line monitoring.

12:15 pm Get More Molecules to the Clinic Faster with Automated Opto™ Cell Line DevelopmentRenee Tobias, Director, CLD Product Management, Marketing, Berkeley Lights, Inc.CHO cell line selection represents a painful bottleneck in biotherapeutic development. The Opto™ CLD workflow on the Beacon® system accelerates CLD by integrating cell enrichment, cloning, culture, productivity and quality screening into a single, automated microscale process that generates the highest titer clones in just 5 days. With in-process detection of product aggregation and FDA-accepted monoclonality assurance, Opto CLD can save valuable development time and help de-risk the path to IND.

12:45 Enjoy Lunch on Your Own





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1:15 Refreshment Break in the Exhibit Hall with Poster Viewing

DEVELOPING STABLE CELL LINES AND REDUCING AGGREGATE FORMATION (VIRTUAL SESSION)

2:00 Speed, Cost, and Quality: Unlocking the Potential of Alternative Hosts for BiologicsChristina S. Alves, PhD, Head, Cell Line Development, BiogenWith the advent of novel genomic editing tools and efficient sequencing techniques, alternative hosts such as eukaryotic microbes have re-emerged as viable expression systems for next generation manufacturing of biologics. Ongoing efforts focused on engineering these organisms have potential to generate a system capable of high productivity, uniform product quality, and short development timelines.

2:30 Using Bioinformatics to Characterize Stable Production Cell LinesSofie O’Brien, PhD, Scientist, Cell Live Development, Seagen, Inc.CHO biologics-producing cell lines must demonstrate consistent growth, productivity, and product quality over the course of manufacturing. To characterize features of stable expression, clonally-derived primary production cell lines and their subclones were studied using high-throughput omics technologies. Differential gene expression analysis identified expression patterns related to differences in clonal heterogeneity, stability, aging, and productivity, identifying genetic pathways which may be useful to improve the clone selection process.

3:00 Reducing Cell-Cell Aggregate Formation and Insulin Degradation in Culture MediaShahram Misaghi, PhD, Principal Scientist, Cell Culture and Bioprocess Operations (CCBO), Genentech, Inc.Cell engineering is used to enhance manufacturing processes and improve titer/product quality in CHO cultures. Here we present two case studies where we investigated 1) role of intercellular cell adhesion molecule-1 (ICAM-1) in cell-cell aggregate formation in CHO cultures and 2) unexpected expression of insulin degrading enzyme (IDE) by CHO cells and its contribution to insulin degradation in culture media.

3:30 Repairing the Repairers: Stabilizing the CHO Genome for Enhanced Protein ProductionNathan Lewis, PhD, Associate Professor, Pediatrics and Bioengineering, University of California, San Diego (UCSD)CHO cells exhibit substantial instability, requiring careful clone selection and stability testing. One mechanism of production instability stems from extensive DNA damage. we identified across several CHO cell lines, DNA damage repair machinery that itself was damaged or suppressed. Following replacement of damaged alleles, we demonstrated increased stability of the CHO genome and increased production stability. Thus, by repairing the repairers, one can stabilize lead clones used for biotherapeutic manufacturing.


4:45 Interactive DiscussionsInteractive Discussions are informal, moderated discussions, allowing participants to exchange ideas and experiences and develop future collaborations around a focused topic. Each discussion will be led by a facilitator who keeps the discussion on track and the group engaged. For in-person events, the facilitator will lead from the front of the room while attendees remain seated. For virtual attendees, the format will be in an online networking platform. To get the most out of this format, please come prepared to share examples from your work, be a part of a collective, problem-solving session, and participate in active idea sharing. Please visit the website’s Interactive Discussions page for a complete listing of topics and descriptions.

IN-PERSON INTERACTIVE DISCUSSION: Impact of Omics, Bioinformatics and Emerging Technologies on CHO Cell Line Stability and DevelopmentDerrick C. Scott, PhD, Associate Professor, Bioinformatics and Executive Director, DSU Molecular Diagnostics Laboratory, Delaware State University

5:45 Close of Cell Culture & Cell Line Optimization Conference





11th Annual

Bioproduction: Scale, Bioreactors & DigitalizationAccelerating Bioprocess & Manufacturing August 18-19

All Times EDT


WEDNESDAY, AUGUST 18


BIOPROCESS DEVELOPMENT UNDER PANDEMIC CONDITIONS

7:55 Chairperson’s RemarksJames V Blackwell, PhD, President & Principal Consultant, Windshire Grp LLC

8:00 KEYNOTE PRESENTATION: 2+1: Tackling the Pandemic Using an Accelerated, Multi-Pronged ApproachDavid Maraldo, PhD, Vice President, Global Vaccines & Biologics Commercialization, Merck & Co., Inc.

Simultaneous acquisition, development, commercialization, and manufacturing readiness of three SARS-CoV-2 biopharmaceutical candidates has fostered innovation, developed new ways of working, and stimulated new technology, all with a singular focus: speed. Use of flexible facilities, modality agnostic platform technologies, and the incorporation of process analytical technology has driven acceleration opportunities. These innovations have created a responsive framework for new product introduction, with principles that have broad, future applicability.

8:30 The Pandemic-Caused Process Changes and Lessons for the FutureJames V Blackwell, PhD, President & Principal Consultant, Windshire Grp LLCThe COVID-19 pandemic has caused significant changes, both disruptions and accelerated development. Lessons learned can inform practices from the production floor to regulatory guidance for the commercialization of future therapeutics and vaccines. This presentation looks at how organizations adapted to the unprecedented critical need to get to market with life-saving vaccines; and how process development; manufacturing/operations/facilities; partnerships; and regulatory change can accelerate development in the future.

9:00 A Single-Use Clinical Manufacturing Facility Operating Under the Realities of Impact from COVIDShawn P Allwein, PhD, Sr Dir Mfg & Operations, Biologics CMC & Specialty R&D, Teva PharmaceuticalsImpact of COVID-19 to the supply chain for raw materials and consumables created major challenges for biopharmaceutical manufacturers. Single-use facilities are by nature heavily dependent on steady supply of consumables. In this presentation, we will describe how proactive planning, strong supplier relationships, creativity to find alternatives, process flexibility and risk tolerance can work to ease the supply constraints and continue to deliver to patient needs.

9:30 CO-PRESENTATION: Process Analytics for Automated Feed Control in Upstream BioprocessingUlrike Rasche, PhD, Scientific Communications Manager, Eppendorf Bioprocess Center, EppendorfWilliam Gonzalez, Bioprocessing Field Application Specialist, EppendorfWhether it be production of antibodies, vaccines, enzymes, or other product, software is needed to monitor and control the process and analyze process data. The capabilities of the software are critical to implement inline process analytics and to implement feedback loops for automated process control. Here we present two strategies for in line process analytics aimed at automated feed control in an upstream bioprocess: Raman analyzer integration and exhaust analyzer integration.


10:40 Bioprocess Development at Pandemic PaceStefan Wieschalka, Scientist, Early Stage Bioprocess Development, Boehringer Ingelheim Pharma GmbH & Co. KGAfter the challenging year 2020, it is evident that rapidly evolving diseases call for bioprocess development at pandemic pace. In the case study at hand, we demonstrated accelerated development of an IgG antibody production process by parallelization of early development work packages and by relying on producer pool fermentations up to Phase 1 clinical supply.

11:10 KEYNOTE PRESENTATION: CMC at the Bill & Melinda Gates Medical Research Institute: Cost to Serve Drives Manufacturing and SupplyHong Liu, PhD, Biologics Process Development and Manufacturing Leader, Bill & Melinda Gates Medical

Research InstituteGates MRI’s mission is: develop products to prevent and treat tuberculosis, malaria, enteric infections and maternal, neonatal and childhood diseases, which are major causes of mortality and inequality in low-middle income countries. Urgency, affordability and accessibility are drivers to minimize cost of manufacturing, reduce time and complexity of supply. CMC makes invaluable contributions by employing technologies to enable first-in-human quickly and also provide clinically viable and commercially attractive by proof-of-concept.

11:40 Cell-Free Plasmid DNA Manufacturing ServiceShigemasa Sasaki, CBO/COO, OriCiro Genomics IncOriCiro® Cell-Free cloning system wipes the bottleneck of conventional E. coli cloning and It just two steps in vitro.OriCiro® ‘s enterprise solution for Cell-Free Plasmid DNA Manufacturing Service has capable to shows in the following benefit. • Rapid : Reaction by several hours, isothermal and enzymatic reaction,• Safety : No endotoxin, No GMO, No antibiotics and antibiotic-resistant

genes and• Process enhancement : Significant downsizing for upstream and

downstream facility






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SCALE-UP AND MANUFACTURING OF AAVs1:25 Chairperson’s RemarksJeffrey Hung, PhD, Chief Commercial Officer, Vigene Biosciences, Inc.

1:30 Design, Manufacturing and Analytics of New AAV Reference Standards – A Case StudyJeffrey Hung, PhD, Chief Commercial Officer, Vigene Biosciences, Inc.AAV Reference materials for gene therapy has been lacking, resulting in poor inter-lab and inter-product comparability. Vigene has developed a standardized production protocol and analytics panel for AAV1, AA2, AAV5, AAV6, AAV8 and AAV9 empty and full capsids to facilitate the testing of AAV gene therapy products. The production process and analytical results will be discussed. Some lessons learned from this year-long endeavor will also be shared.

2:00 Optimisation and Scale-Up of Suspension-Based Manufacturing Processes for AAV ProductionHelen Young, PhD, Senior Scientist II, Advanced Therapeutics, CPIDeveloping high-yielding, robust, scalable and commercially viable processes to manufacture sufficient quantities of viral vectors presents challenges. This work focused on development of two suspension-based AAV production processes, scaled from shake flasks to ambr250, 10L, 50L and 200L stirred tank bioreactors. Scale-up presented challenges yet through high-throughput optimisation utilising a DoE approach, performed using the ambr250, high-yielding and scalable processes were developed with titres maintained up to 200L scale.

2:30 Scaling AAV and Cell Production to Manufacturing Scales with a Novel Intensified Adherent Cell Culture SystemJohn Yoshi Shyu, PhD, Director, Scientific Applications and Technical Support, Americas, Corning Incorporated - Life SciencesPressure to reduce time-to-market and production cost of viral vectors and cell-based therapeutics is driving demand for technologies that combine the greater yield efficiency and viable cell harvest of adherent platforms with the scale and automation of suspension systems. Dr. Shyu will present a new intensified FBR technology that has demonstrated a multi-fold increase in viral vector yield per cm2 and greater than 90% transfection rate and cell harvest efficiency.


PLENARY KEYNOTE SESSION: LEADING TO TOMORROW’S ADVANCES

3:50 Plenary Keynote IntroductionDominic Clarke, PhD, ISCT Process & Product Committee Co-Chair & Chief Technology Officer, Cell and Gene Therapy, Discovery Life Sciences

4:00 KEYNOTE PRESENTATION: Manufacturing Next-Generation TherapiesMartha Rook, PhD, Chief Technical Operations Officer, Sigilon Therapeutics, Inc.Cell and gene therapies have shown dramatic clinical

progress in recent years. Driven by this clinical success and the needs of commercialization, manufacturing strategies are progressing. Nevertheless, a standard manufacturing template for these therapies has not evolved and with novel modalities continuing to emerge we may be in a post-template manufacturing landscape. CMC strategies must be developed to avoid manufacturing becoming a roadblock to therapeutic success.

4:30 KEYNOTE PRESENTATION: Sustainability and the Future of BioprocessingKristi Budzinski, PhD, Principal Product Stewardship & Green BioPharma Manager, Genentech, Inc.The future of bioprocessing demands flexible, scalable

solutions that can accommodate the rapidly evolving landscape of biopharmaceutical products while also minimizing impact on the environment. This talk will highlight some of the major opportunities for reducing the environmental impact of bioprocessing through the application of metrics such as process mass intensity and lifecycle assessment methodology. Results will be presented from both a Genentech perspective and industry-wide perspective.

5:00 Networking Reception in the Exhibit Hall with Poster Viewing

6:00 Close of Day

THURSDAY, AUGUST 19


INNOVATIONS IN BIOPRODUCTION I - BIOREACTORS, GREEN BIOTECH, MACHINE LEARNING AND DIGITAL

TWIN (VIRTUAL SESSION)8:00 Implementation of Custom Single-Use 2L Bioreactors in Upstream Process DevelopmentBalrina Gupta, PhD, Associate Principal Scientist, Biological & Sterile Product Development, Merck Research LabsEvolution of 2-L single-use bioreactor design customization and assessment, with performance comparison to traditional glass bioreactors, will be presented. Multiple cell lines, different media and modes of operation were examined to support implementation of single use across pipeline projects.





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8:30 Scale-Down of a Microbial Fermentation Process to Bottles and 250mL Single Use Bioreactors to Enable Collection of Process Characterization DataMatthew Woodling, Associate Principal Scientist, Vaccine Bioprocess R&D, Merck & Co Inc.Bioprocess development requires multiple process scales to enable the collection of large data sets that represent the commercial scale process of the product. This presentation reviews the scale down of a microbial fermentation process to the Sartorius Ambr 250mL single use bioreactor system and the associated time and cost savings when generating process characterization data at this smaller scale.

9:00 am Coffee Break in the Exhibit Hall with Poster Viewing

9:30 Application of Machine Learning Methods to Pathogen Safety Evaluation in Biological Manufacturing ProcessesShyam Panjwani, PhD, Senior Data Scientist, Bayer Healthcare PharmaceuticalsThe production of human monoclonal antibodies is not free of viral contamination risk due to adventitious agents in raw materials, cell substrates and environment. Such risk is generally mitigated by a series of time and resource expensive unit operations. Machine learning methods can potentially help streamline the development and optimization of viral clearance steps. The evaluation of machine learning methods for viral clearance operations is the focus of this study.

10:00 Digital Twin for Biotech ProcessesSandrine Dessoy, Senior Manager, Tech R&D, GlaxoSmithKlineA digital twin is a real-time digital replica of a physical device that communicates with it. A digital twin predicts the evolution of its corresponding physical asset and to reach this goal it utilizes state-of-the-art mechanistic and/or hybrid models, advanced sensors, connected objects and artificial intelligence. This talk is concerned with the development of digital twins in GSK for development and control of vaccines production process.

10:30 Green Biotechnology with Microalgae: Always Almost ThereJulian Rosenberg, PhD, Associate Director, Center for Biopharmaceutical Education & Training (CBET), Albany College of Pharmacy & Health SciencesMicroalgae are versatile photosynthetic organisms poised to become a sustainable platform for therapeutics, vaccines, biofuels, and plant-based protein. However, bioprocessing challenges continue to limit production at commercial scales. While the field has benefitted from advances in bioreactor design, strain selection, and bioengineering, the viability of algae-based products seems to always be just out of reach. This presentation reviews R&D accomplishments in algal biotechnology and identifies major hurdles to successful commercialization.

11:00 Interactive DiscussionsInteractive Discussions are informal, moderated discussions, allowing participants to exchange ideas and experiences and develop future collaborations around a focused topic. Each discussion will be led by a facilitator who keeps the discussion on track and the group engaged. For

in-person events, the facilitator will lead from the front of the room while attendees remain seated. For virtual attendees, the format will be in an online networking platform. To get the most out of this format, please come prepared to share examples from your work, be a part of a collective, problem-solving session, and participate in active idea sharing. Please visit the website’s Interactive Discussions page for a complete listing of topics and descriptions.

IN-PERSON INTERACTIVE DISCUSSION: Pushing the Envelope in Bioprocess Development – From Disposables, Continuous Manufacturing to Bioprocessing-in-a-ContainerDavid W. Wood, PhD, Professor, Chemical & Biomolecular Engineering, The Ohio State UniversityPotential topics for discussion:• What cutting edge technologies are likely to change the industry in the

next 10 years?• What sorts of big ideas have been enabled by these new technologies,

beyond the obvious applications?• What problems are likely to remain, which will require additional disruptive

innovations to solve?• How can a company balance vision with risk when making bold steps

forward?


12:00 pm Refreshment Break in the Exhibit Hall with Poster Viewing

INNOVATIONS IN BIOPRODUCTION II - MODELING, AUTOMATION & SINGLE-USE SYSTEMS (IN-PERSON

SESSION)12:35 Chairperson’s RemarksEugene Tung, PhD, Executive Director, Manufacturing IT, Merck & Co., Inc.

12:40 Mechanistic Modeling and Parameter-Adaptive Nonlinear Model Predictive Control of a MicrobioreactorMoo Sun Hong, PhD, Postdoctoral Associate, Chemical Engineering, Massachusetts Institute of TechnologyA mechanistic model is presented for the spatiotemporal transport of oxygen through a gas-permeable membrane to the cells within a microbioreactor. The model is used to design an on-line estimator for the oxygen uptake rate of the cells, the specific cell growth rate, and the specific oxygen uptake rate. The estimates are fed to a model predictive control formulation showing improved spatial control of dissolved oxygen during cell growth.





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1:10 Development of a Practical Approach for Evaluation of Single Use Fermenters with Application in Diagnostic IndustryYongxue Ding, PhD, Principal Scientist, Biologics Process Design R&D, Abbott Diagnostics Division, Abbott LaboratoriesWe will present a method through generating the ratio (%) of k_L a alike between SIP and SUF. Two scenarios were analyzed, one at low demand standard density growth, and the other at high density growth. The results matched with performance of SUF to SIP very well. This enables a practical approach for evaluation of SUF as replacement of SIP for production of biologics in diagnostic business.

1:40 Plug and Play Automation for BioprocessingEugene Tung, PhD, Executive Director, Manufacturing IT, Merck & Co., Inc.The BioPhorum Plug and Play Standard was conceived and developed through an industry-wide collaboration to enable the rapid deployment of modular equipment in biopharmaceutical facilities. The standard, co-developed by equipment providers, automation suppliers, and end-users, provides a common structure for defining an equipment’s automation capability. It utilizes existing and proven industry communication standards, eliminating the need for custom communication interfaces. Eliminating custom interfaces can save weeks in deploying automated equipment.

2:10 Refreshment Break in the Exhibit Hall & Last Chance for Poster Viewing

INNOVATIONS IN BIOPRODUCTION III - DIGITALIZATION, REAL-TIME MONITORING & PAT

(VIRTUAL SESSION)2:50 Real-time Monitoring of AAV Production Culture System by Digital Differential Holographic MicroscopyDainan Mao, PhD, Scientist II, Ultragenyx PharmaceuticalBesides conventional offline analytical testing for AAV production at bench scale, a digital differential holographic microscopy system was developed to monitor suspension cell culture systems. Key process parameters including viable cell density and transfection/infection efficiency were determined at real-time for the production process. This process analytical technology (PAT) holds promise for future upstream process optimization for gene therapy products.

3:20 Automating Upstream: Implementing Advanced Process Control, Predictive Modelling and Online PAT Tools in Mammalian Cell Culture ProcessesBethany Kerr, Team Leader - Upstream Development, CPIAn improved understanding of the culture environment within bioreactors is essential for developing robust, high yielding processes. We will explore the usage of advanced process control and predictive modelling in mammalian cell cultures to automate and optimise bioreactor feeding and control strategies. A series of case studies will cover topics including utilising integrated PAT tools for automated at-line feeding and using machine learning to control and refine bioreactor set points.

3:50 Digitalization, Data Enablement and Analytics for Accelerated Bioprocess DevelopmentJun Huang, PhD, Senior Director, Data Enablement and Analytics, Regeneron PharmaceuticalsDigitalization, data enablement and analytics are key drivers to accelerate bioprocess development. In this talk, the operationalization of our digital/data strategy through a structured and agile program delivery will be discussed, covering 1) unified data management and IT/OT infrastructure to improve data access, sharing and integration, and 2) analytics and control capabilities to increase process visibility, comparability and predictability, as well as productivity and quality.

4:20 Close of Conference





Downstream Downstream ProcessingProcessingNew and complex molecules such as oligonucleotides, AAVs, ADC, fragments, vaccines, and others coming down the pipeline are continuing to challenge the downstream purification and recovery process. Companies are looking toward disruptive innovations and creative strategies, including integrated/continuous processing, improvements in upstream perfusion, process integration and optimization, as well as latest advances and breakthroughs in filtration, clarification, capture and purification. Join our Downstream Processing to hear these exciting approaches and share best practices for downstream purification of these new modalities.

Advances in Purification

& Recovery

Continuous Processing in Biopharm

Manufacturing

Conference Programs

AUGUST 16-17

AUGUST 18-19






7th Annual

Continuous Processing in Biopharm ManufacturingNew Advances and Novel Approaches

August 16-17All Times EDT


MONDAY, AUGUST 16


CONTINUOUS BIOMANUFACTURING CASE STUDIES 9:55 Chairperson’s RemarksAlois Jungbauer, PhD, Professor & Head, Biotechnology, Institute of Bioprocess Science and Engineering, University of Natural Resources and Life Sciences (BOKU)

10:00 KEYNOTE PRESENTATION: Scale-Up of Continuous Antibody Manufacturing Process from Bench to Pilot Scale and BeyondAlex Brinkmann, Senior Engineer II, Biopharmaceutical Development, Biogen

We will share our experience with scaling up a continuous manufacturing process from bench to pilot scale, as well as progress towards an engineering run. We will focus on the challenges associated with integration of the perfusion bioreactor to the capture skid via a novel application of SPTFF to enhance capture productivity, and pooling strategies for the polishing unit operations, as they relate to process cadence and product quality.

10:30 A Hybrid Approach to Continuous Biomanufacturing of BiosimilarsAndrew Falconbridge, Vice President, Process Technology & Innovation, AlvotechThere has been an increasing trend in developing unit operations that can been utilised in a continuous fashion, although fully continuous end to end operations are not fully realised yet there are opportunities to use these unit operations in a hybrid mode to increase output, reduce footprint and reduce cost of goods. Data from experiments to show effectiveness of these approaches.

11:00 Successful Scale Up of an Intensified Perfusion Process to Clinical and Commercial ScalesGabriel Lurz, PhD, Research Engineer, SanofiAn intensified perfusion process for production of a therapeutic monoclonal antibody was developed and scaled to 100 L clinical and 500 L commercial scales. The baseline process was developed in 10 L bench top bioreactors with stainless steel alternating tangential flow (ATF) cell retention systems. These studies demonstrate that intensified perfusion processes developed in benchtop bioreactors can be successfully reproduced at scales relevant for manufacturing.

11:30 Automation of an End-to-End Continuous Purification Process with the Pilot PAK SystemKevin Hill-Byrne, Vice President, PAK BioSolutionsPAK BioSolutions newly released system performs end-to-end continuous purification of biopharmaceuticals in a fully-automated manner and is highly configurable to match any purification process design. The systems capabilities were demonstrated with continuous purification of a 50L perfusion bioreactor over 14 days. We will present product quality and bioburden data from capture chromatography, virus inactivation, filtration, and polishing membrane operations performed in series simultaneously with a single piece of equipment.

11:45 Using Real-time Monitoring and AI for Continuous Process OptimizationAndy Alasso, Senior Vice President of Product Management, AizonComplexity grows when we move from batch to continuous processing and a unified platform ensures you have the right tools for real-time processing, optimizing processes, and predicting yields to produce the best quality product. Learn how to: • Generate insights by bringing in data real-time from disparate sources• Effectively adopt and integrate AI in a qualified manner• Find patterns that may lead to deviations and take preventative action• Future-proof for what’s ahead


ECONOMICS, INTEGRATION & OPTIMIZATION OF CONTINUOUS PROCESSES

12:50 Chairperson’s RemarksAlois Jungbauer, PhD, Professor & Head, Biotechnology, Institute of Bioprocess Science and Engineering, University of Natural Resources and Life Sciences (BOKU)

12:55 Process Integration for Up and Downstream ProcessingAlois Jungbauer, PhD, Professor & Head, Biotechnology, Institute of Bioprocess Science and Engineering, University of Natural Resources and Life Sciences (BOKU)Continuous integrated manufacturing can be achieved of biopharmaceuticals has been achieved by converting batch unit operations into a pseudo continuous operation. Here we demonstrate a real, fully continuous operation and a monitoring and control concept for production of antibodies. The perfusion culture is directly linked to a continuous precipitation and virus inactivation in a single line. The economic advantages for such a process will be also discussed.

1:25 Continuous Biomanufacturing Platform and Regulatory RequirementsRobert F. Dream, PhD, Managing Director, HDR Co. LLCContinuous Biomanufacturing is the highest level of integrated bioprocessing to deliver drug products. These qualities enable unique functionality and platforms capable of rapid deployment delivering agile and accelerated timelines from development to on-demand manufacturing. The drivers for adoption of CBM relate to improved productivity, reduced plant footprint, and favorable lifecycle considerations. These come at the cost of more sophisticated supervisory controls, to enable consistent quality and in-process continued verification.

1:55 Sponsored Presentation (Opportunity Available)






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2:40 Optimising the Intensified Seed Train: An Economic PerspectiveAndrew Sinclair, President & Founder, BioPharm Services Ltd., United KingdomThis talk will focus on the upstream assessing the impact of strategies for process intensification:• Concentrated N-1 seed, perfusion, what are the optimum configurations• High turndown bioreactors in the seed train• Impact of concentrated Fed batch• The implications of deploying large volume single-use bioreactors, upto

6000LThe outcomes of modelling will provide insights into the benefits and the issues that need to be addressed for a successful financial outcome.

3:10 From Tap to Waste – Water Dedicated CO2 Evaluation Using the WARIEN MetricAlessandro Cataldo, PhD, Researcher, Former - University of Natural Resources and Life SciencesManufacturing of biopharmaceuticals requires high quality water. We combined economic and ecological modeling to assess energy consumption of water production from tap to waste and defined the WAter Related Impact of ENergy (WARIEN) metric to directly correlate the amount of CO2 emitted per kg biopharmaceutical. The water related costs per kg product and the WARIEN correlate and therefore can be used as a design criterion for biopharmaceutical process development.









6:30 Close of Day

TUESDAY, AUGUST 17


IMPROVING UPSTREAM PERFUSION PROCESS7:55 Chairperson’s RemarksJongyoon Han, PhD, Professor, Electrical Engineering & Computer Science, Massachusetts Institute of Technology

8:00 Mathematical Modelling of a CHO Cell Perfusion ProcessVeronique Chotteau, PhD, Coordinator of iConsensus, Industrial Biotechnology, KTH Royal Institute of TechnologyMathematical model of process can help for process simulation, prediction, monitoring and/or control. We have developed modelling methodologies suitable for perfusion or fed-batch processes, including amino acid metabolism or glycosylation. We will illustrate the prediction power of these models in high cell density perfusion runs. The models are based on the measurements of the extracellular metabolites, and include an objective and automatic determination of the used pathway and the kinetics.

8:30 Online Capacitance Controlled Perfusion N-1: Optimization of Media Usage and Process Development FlowEmily Rittershaus, PhD, Scientist, Process Development Upstream, Bristol-Myers Squibb Co.

9:00 An Update on Cell Line Development for Lentiviral Vector Production in Perfusion ModeAziza Manceur, PhD, Research Officer, National Research Council CanadaA perfusion process is particularly well suited to the biomanufacturing of lentiviral vectors due to their labile nature. A comparison of the titers obtained in different modes of operation will be summarized. We favor the use of stable producer cell lines over transfection, and we demonstrate that generating the most stable cell line possible is worthwhile. Finally, our efforts towards a semi-continuous process from upstream to downstream will be presented.





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9:30 PendoTECH Single Use Sensors: Proven Technology for Existing Single Use Processes and Future-Ready for BioProcessing 4.0Jim Furey, General Manager & Marketing Manager, PendoTECHFor over 10 years, PendoTECH Single Use Sensors have been used extensively both in the laboratory for process development and in GMP processes to enable elimination of cleaning and facilitate rapid turnover of process equipment. Their primary applications are for measurement of pressure, temperature, conductivity, UV, and turbidity in a range of upstream and downstream applications. PendoTECH is offering a platform to enable all of its sensors to smoothly transition to use for Bioprocessing 4.0.


CONTINUOUS PROCESS MONITORING AND CONTROL10:45 Continuous Online Protein Quality Monitoring during Perfusion Culture Production Using Integrated Micro/Nanofluidic SystemJongyoon Han, PhD, Professor, Electrical Engineering & Computer Science, Massachusetts Institute of TechnologyWe demonstrate a new micro/nanofluidic system for continuous and automatic monitoring of protein product size and quantity directly from the culture supernatant during a high-cell-concentration CHO cell perfusion culture. The continuous-flow and fully automated operation of this nanofluidic protein analytics reduces design complexity and offers more detailed information on protein products than offline and batch-mode conventional analytics.

11:15 Multi-Attribute Chromatography for Bioprocess MonitoringHelen Zhao, M.S., Scientist, Process Development Analytics, Bristol-Myers Squibb Co.On-line Multi-attribute Chromatography (MAC) is one of the PAT tools that allows measurement of few critical quality attributes in real time. Here we describe an On-line MAC platform featuring Two-Dimensional Liquid Chromatography (2DLC) and automated-sampling device for real time monitoring of charge variants and aggregation in production bioreactor. This technique can be applied for monitoring from multiple bioreactors with different protein modalities, and and potential for analysis of charge variants.

11:45 Automated Identification, Adaption and Deployment of Digital TwinsChristoph Herwig, PhD, Head Research Area Biochemical Engineering, TU ViennaDigital twins will play crucial role in continous biomanufacturing, because product quality needs to ensure in a time continuum. The digital twins need to be set up and also maintained in an efficient but also robust manner, so that they keep up with the evolution of the process life cycle. This contirbution will show new workflows how to generate and adapt digital twins for upstream and downstream applications.

12:15 pm Monitor Protein Concentration in Real Time with Pall’s Inline Index of Refractivity (IoR) SystemJulio Huato, Research Scientist, Bioprocess Research and Development, Pall CorporationPall’s mPath IoR concentration monitor provides accurate inline continuous measurement of product concentration in bioprocess fluids, enabling more rapid development and characterization of biotech processes. These measurements enable scientists to quickly see the impact of process changes on product yield, allowing fast determination of optimal process parameters



NOVEL APPROACHES AND NEW ROUTES IN CONTINUOUS BIOLOGICS PROCESSING

1:55 Chairperson’s RemarksStefano Menegatti, PhD, Assistant Professor, Chemical & Biomolecular Engineering, North Carolina State University

2:00 Continuous Clearance of Host Cell Proteins from CHO, HEK, and Pichia Harvests: A Novel Route to Purify Proteins and Viral Vectors for Gene TherapyStefano Menegatti, PhD, Assistant Professor, Chemical & Biomolecular Engineering, North Carolina State UniversitymAb purification must ensure the removal of host cell proteins (HCPs), endotoxins, mAb fragments and aggregates. Commercial adsorbents can struggle to remove particular “high-risk” HCP impurities that pose direct threat to health. We demonstrated a novel adsorbent – LigaGuardTM – that captures HCPs in flow-through mode and enables continuous mAb purification from CHO, HEK, and Pichia fluids. We demonstrated >92% recovery, >95% purity, HCP-LRV ~ 2, and clearance of “high-risk” HCPs.

2:30 Biofilm Reactors: A New Horizon in Continuous Production of Recombinant BiopharmaceuticalsEhsan Mahdinia, PhD, Assistant Professor, Stack Family Center for Biopharmaceutical Education & Training, Albany College of Pharmacy & Health SciencesMicrobes in biofilm forms usually express magnificent potentials for boosted product secretion through profound genetic alterations during the immobilization process. Biofilm reactors provide the controlled environments for filament and biofilm formers to express these great advantages without the associated transport phenomena shortcomings and scale-up issues.

3:00 Development of Novel Dynamic Perfusion Methodology with Compartmentalized Media Concentrates for Next-Generation Continuous BioprocessingDr. Yiu-Sun Hung, Scientist II, Cell Culture - Process Science, Boehringer IngelheimWe have developed an osmolality-based dynamic perfusion process utilizing compartmentalized media concentrates to support ultra-high cell density (>100e6 cells/mL). This process eliminates the need for cell bleeds





7th Annual




since it can achieve substantial productivity improvement in the length of conventional mammalian upstream process. Pilot-scale application further demonstrates a 10-fold enhancement in productivity compared with fed-batch operations, revealing its potential as an innovative approach for next-generation continuous manufacturing.

3:30 Evaluation of the Erbi BreezTM Microbioreactor SystemJared Franklin, Principal Research Associate, Medium & Bioprocess Technologies, Sanofi USThe 2 mL Erbi Breez micro-bioreactor system (developed by Erbi Biosystems, Woburn, MA) utilizes microfluidic technology to integrate the reactor chamber, pump mechanism, process sensors, and perfusion unit operations into one single-use cassette. The small footprint, system flexibility, and integrated controls make it a promising candidate for an intensified perfusion scale-down model. Our primary evaluation generated results that are comparable to the bench scale perfusion process.



IN-PERSON INTERACTIVE DISCUSSION: Novel Approaches in Continuous Biologics ProcessingEhsan Mahdinia, PhD, Assistant Professor, Stack Family Center for Biopharmaceutical Education & Training, Albany College of Pharmacy & Health Sciences• What are the major breakthroughs in upstream and downstream

processing; current or upcoming, in enabling the adaptation of continuous bioprocessing?

• N-1 perfusion columns in upstream; Hollow fiber filters in downstream; Cell carriers in fixed-bed or basket bioreactors; Biofilm fermenters; Inline process analytical tools; Quality by design; Modeling tools.

• What are the interactions between single-use technologies being adapted and replacing conventional stainless steel infrastructures in biopharma with continuous bioprocessing strategies??

5:45 Close of Continuous Processing in Biopharm Manufacturing Conference





7th Annual

Advances in Purification & RecoveryRising to the Challenge of New and Complex Modalities August 18-19

All Times EDT




NOVEL APPROACHES AND NEW BREAKTHROUGHS IN SEPARATION, PRECIPITATION AND FILTRATION

(VIRTUAL SESSION)

8:00 KEYNOTE PRESENTATION: New Breakthroughs in Protein Separation: High Capacity Magnetic Adsorbers and Their Automated Magnetic SeparationSonja Berensmeier, PhD, Professor, Bioseparation

Engineering Group, Mechanical Engineering, Technical University of MunichWe show two examples of biocompatible low-cost magnetic nanoparticles (MNP). First, we show new peptide tags and their fusion proteins that bind highly selectively to the bare MNPs. Second, we demonstrate the potential of Protein A functionalized MNP for antibody purification on the example of enormous capacities. Finally, the newest status of an automated high-gradient magnetic separator system in the technical scale is presented.

8:30 Development and Process Characterization of a Precipitation Step Using Sodium Caprylate for the Reduction of Host Cell ProteinsJessica Prentice, Purification Process Sciences, BioPharmaceutical R&D, AstraZeneca, Gaithersburg, USWe describe the development and characterization of a precipitation step using sodium caprylate for the reduction of host cell proteins during monoclonal antibody purification. Development and process characterization utilized Quality by Design principles in an efficient, streamlined two-step approach to provide understanding of process parameter impacts and process robustness. Data is presented to illustrate the development, characterization and robust reduction of host cell proteins using sodium caprylate precipitation.

9:00 Progress on Continuous Process Development Toward the Envisioned Next-Generation Factory ConceptTomonori Shiotani, CMC Researcher, API Process Dev, Chugai Pharmaceutical Co LtdWe aim to develop highly productive integrated continuous downstream processes to realize our envisioned next-generation factory concept where extremely low manufacturing cost would be achieved along with environmental friendliness. In this presentation, our future vision and recent highlighted progress will be reported: (1) next-generation factory concept and facility design status, (2) long-duration capture MCC operation connected with perfusion upstream, (3) scale-down demonstration run of integrated filtration-polishing.

9:30 CO-PRESENTATION: Reflections on the Past and Future of Downstream BioprocessingAkunna Iheanacho, PhD, Scientific Director, Texcell-North America, Inc.Brandy Sargent, Editor in-chief Cell Culture Dish & Downstream Column, Cell Culture Dish


10:40 Revolutionizing the Downstream Processing of Monoclonal Antibodies by Continuous Template-Assisted Membrane CrystallisationSean Ruane, PhD, Senior Scientist, Process Automation, CPIThe AMECRYS project is an EU-funded consortium with the ambitious goal of producing a disruptive alternative to traditional chromatography-based mAb purification, by forming mAb crystals directly from cell culture material. This presentation will cover the background and later stages of the project, focusing on CPI’s role in developing mAb crystallization at process scale.

11:10 A Novel Pirani-Flowrate Correlation Soft-Sensor to Enable PAT-Based Lyophilization Drying Endpoint Determination and OptimizationTong Zhu, PhD, Senior Scientist, New Biological Entities (NBE) Pharmaceutical Sciences, AbbVie, Inc.Full convergence of Pirani to capacitance manometer pressure was traditionally used to determine the endpoint of primary drying, which is usually inefficient due to lacking key understanding of the correlation between Pirani pressure (or moisture history) and the underlying vapor flow rate. The present study uses computational fluid dynamics (CFD) modeling to build a soft-sensor PAT tool to predict this correlation and enable safe reduction of the lyophilization process time.

DOWNSTREAM PROCESSING OF VIRAL VECTORS (VIRTUAL SESSION)

11:40 Recovery of Viral Vectors with Nanofibre ChromatographyDaniel G. Bracewell, PhD, Professor Bioprocess Analysis, Biochemical Engineering, University College LondonViral vector processes are still maturing. Here we study adenovirus and lentivirus. The labile nature of these products is a concern when using adsorption-based separation. Downstream processing sequences involving hollow fibre UF/DF and nanofiber based ion exchange chromatography were investigated. Minimising the time spent in the adsorbed state was shown to be beneficial in retaining high yield. While resolution is highly sensitive to the ion exchange ligand density.



1:30 Scalable Downstream Process Development and Scale-up for in-vivo Gene Therapy ProductsKumar Dhanasekharan, PhD, Vice President, Technical Operations, SwanBio TherapeuticsMonolith-based ion exchange chromatography (IEX) is a promising tool to achieve this separation but poses challenges with consistency upon scale-up. This paper outlines some of the scale-up challenges and potential mitigations to achieve consistent separation of full and empty particles. IEX is evolving to become the industry standard for downstream separations as industrialization of gene therapy manufacturing continues.





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2:00 Host Cell Protein Challenges in the Downstream Process Development of Non-Antibody ProcessesNaveenkumar Singh, PhD, Scientist II, Downstream Process Development, Ambrx, Inc.In this example, HCP reduction challenges were encountered with a recombinant protein conjugate expressed in CHO cells. A baseline process using conventional chromatography and filtration techniques was rapidly developed to support Toxicology and Phase 1 production. However, HCP levels of 10,000-20,000 ng/mg were measured at the intermediate stage (pre-conjugation) of the process. Here we discuss the systematic approach to reduce HCP levels to within specification using strategies amenable to manufacturing.

2:30 CO-PRESENTATION: Understanding Monoclonal Antibody Aggregate Behaviour Impacting Chromatographic Process EfficiencyCourtney O’Dell, MS, Senior Scientist, AvantorSuman McLinden, Senior Scientist, AvantorAs titer from upstream increases, capacity of the resins is more of important factor to drive process efficiency and impurities like aggregate reduces the capacity of monomer significantly. In this presentation, we will show the impact of aggregate on the capacity and efficiency of chromatography steps such as protein A, ion-exchange or HIC (hydrophobic interaction) columns along with use of additive and selective resins to increase product purity and process efficiency.









6:00 Close of Day

THURSDAY, AUGUST 19


PURIFICATION APPROACHES FOR NOVEL MODALITIES

7:55 Chairperson’s RemarksEngin Ayturk, PhD, Senior Director, CMC Process Engineering & BioConjugation, Mersana Therapeutics

8:00 Using DoE Models in Process Development of Antisense OligonucleotidesArmin Delavari, PhD, Scientist I, Technical Development, BiogenPurification processes of oligonucleotides are often developed through comprehensive small-scale studies which relies highly on experimental design and interpretation of data. Design of experiments is a statistical tool that provides valuable information for study design, screening and optimization via analyzing impacts of individual and interactive effects of process parameters on process performance. In this presentation, case studies will be discussed where DoE models were used for process intensification and simplification.

8:30 Targeted Therapies via Novel Antibody Drug Conjugate (ADC) Technology PlatformsEngin Ayturk, PhD, Senior Director, CMC Process Engineering & BioConjugation, Mersana TherapeuticsMersana Therapeutics is a clinical-stage biopharmaceutical company using its differentiated and proprietary ADC platforms to rapidly develop novel ADCs with optimal efficacy, safety and tolerability to meaningfully improve the lives of people fighting cancer. This presentation will provide an overview of Mersana’s novel ADC platforms and discuss the QbD-based process development, scale-up and process intensification strategies, leveraging the synergies between synthetic chemistry, small molecule and antibody production, and bioconjugation processes.






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9:30 Purification Approaches for Aggregation-Prone Antibody FragmentWan-Ching Lai, PhD, Principal Research Scientist, Abbott LabsWith the constant improvements to genetic engineering the production of recombinant Fabs has become a viable alternative to antibodies. We previously established a single IMAC step, generating His-Tag rFab with 97% monomer. The IMAC method, however, causes aggregation of another His-Tag rFab and reduces assay potency. The goal is to generate stable purified His-Tag rFab meeting the assay potency requirement. DOE is used to establish the manufacturing purification method.

10:00 Consistent Scalability of High Flow, High Binding Protein A Chromatography Membranes for BioprocessingWilliam Barrett, PhD, Product Specialist, W.L. Gore & AssociatesThe promise of high flow rates offered by affinity chromatography membranes has been limited by insufficient binding capacity, inconsistency, and unproven scalability for process development and manufacturing. Data will highlight flexibility of binding capacity versus residence time and demonstrate consistent scalability at low pressure drop aligning with existing systems and providing a path to developing rapid cycle protocols for GMP clinical manufacturing

10:30 Rapid, Analytical Chromatographic Isolation and Purification of Vectors from Culture Media: Exosomes and LentivirusR. Kenneth Marcus, PhD, Professor, Chemistry, Biosystems Research Complex, Clemson UniversityOur laboratory has developed the use of fiber-based chromatographic stationary phases that allow rapid (<10 min) separations of exosomes and lentivirus particles from cell culture media using standard laboratory HPLC instrumentation. Fibers allow isolation from culture media components and host cell proteins at >95% efficiencies on sample volumes from 10-1000 microliters. Scale-up is also anticipated.






forward?



PURIFICATION APPROACHES FOR NOVEL MODALITIES (HYBRID)

12:35 Chairperson’s RemarksDavid W. Wood, PhD, Professor, Chemical & Biomolecular Engineering, The Ohio State University

12:40 Rapid “Fit for Purpose” Process Monitoring Accelerates Process Screening and Optimization During Early Phase Development of non-mAb TherapeuticsJoseph Kutzko, Principal Engineer, Downstream Development, Drug Substance, MSAT, Sanofi GenzymeIn this presentation, two 3-minute, rapid HPLC methods were developed for their utility in designing downstream processes. The analytics described herein were optimized and employed to develop a purification process for a non-mAb related product. As an added benefit the development timeline was greatly streamlined. This strategy for purification development can be applied to other projects as well.

1:10 Crystallization of a Rotavirus Subunit Vaccine CandidateRichard D. Braatz, PhD, Edwin R. Gilliland Professor, Chemical Engineering, Massachusetts Institute of TechnologyA significant proportion of the cost of manufacturing recombinant protein subunit vaccines is the purification which occurs in multiple chromatography steps. Crystallization has the potential to reduce purification costs and provide new product storage modality, improved operational flexibility, and reduced facility footprints. This presentation describes an automated controlled microdrop-based crystallization system for the collection of thermodynamic and kinetic data and its application to a rotavirus vaccine candidate.





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1:40 Developing a Continuous Capture Process for Recombinant Antibodies Based on PrecipitationGregory Dutra, PhD Student, Department of Biotechnology, BOKUCombining a continuous recombinant antibody precipitation with tangential flow filtration allows for a constant mass flow capture process. Deciding the conditions for the process is an important step to ensure process key parameters that has a direct impact on the purity and recovery rates. We describe what to account for when designing such a process showing a case study with different recombinant antibodies.







Analytical Analytical & Quality& QualityThe Summit’s 2021 Analytical & Quality stream offers in-depth updates on critical steps in biopharmaceutical development that impact product quality, safety, and regulatory compliance. Separate two-day meetings in this stream will focus on the detection, analysis and removal of host cell proteins and the acceleration of analytical development steps and timelines. Over thirty in-depth presentations will give attendees insight into the best practices being applied across large and small industry R&D groups.

Accelerating Analytical Development

Host Cell Proteins

Conference Programs

AUGUST 16-17

AUGUST 18-19






6th Annual

Host Cell ProteinsStrategies and Technologies for Detection, Analysis and Control August 16-17

All Times EDT


MONDAY, AUGUST 16


PROFILING AND CONTROLLING HIGH-RISK HCPs9:55 Chairperson’s RemarksKent Simmons, Senior Conference Producer, Cambridge Healthtech Institute

10:00 KEYNOTE PRESENTATION: Host Cell Protein Characterization for Bioprocess Development: Current Approaches and ChallengesKrishnan Sampathkumar, PhD, Executive Director,

BioPharmaceutical Development, MacroGenics, Inc.Residual Host Cell Proteins (HCPs) in therapeutic biologic products have the potential to affect product quality and immunogenicity in patients. Well-designed assays using reagents with adequate coverage for the specific HCPs needs to be considered for defining the process and acceptance criteria. An overview of approaches for profiling and controlling HCPs, and related issues and challenges observed for antibody-based products using CHO platforms will be presented as case studies.

10:30 Methods for the Robust Quantification of Trace-Level Host Cell Protein Impurities in Antibody Drug ProductsAlain Beck, PhD, Senior Director, Biologics CMC and Developability, Pierre Fabre, FranceWhile overall HCP levels are usually monitored by ELISA, MS-based approaches have been emerging as alternative providing qualitative and quantitative information. However, a major challenge for LC-MS-based methods is to deal with the wide dynamic range of DPs and the sensitivity required to detect trace-level HCPs. Reproducible MS-based analytical workflows coupling optimized and efficient sample preparations, the library-free data-independent acquisition (DIA) method, and stringent validation criteria will be discussed.

11:00 BioPhorum Development Group (BPDG) HCP Working Stream UpdateFengqiang Wang, PhD, Principal Scientist, Analytical Method Development, Merck & Co., Inc.A working stream consisting of 26 member companies initiated a collaboration among its members to align industry best practices and generated a generic risk assessment tool to manage HCP-related risks identified during biologics development from both an assay development and process development perspective. A sub team within the HCP working stream also compiled a list of “high-risk” HCPs from CHO-produced biologics through literature review and cross-company discussions.

11:30 Accelerating Bioprocess Workflows with High-Throughput, Automated Platform for Impurities and Titer AnalysisRob Durham, PhD, Director, Service and Scientific Support, Gyros Protein TechnologiesHost cell protein and other process-related impurities levels are critical quality attributes in biotherapeutic manufacturing. Gyrolab® systems perform automated immunoassays within nanoliter-scale microfluidic structures in Compact Disk format, increasing throughput, the sample

and reagent volumes dramatically as compared to plate-based ELISA. We present case studies showing titer and impurities analytics of mAb bioprocess samples, CHO cells, and adeno-associated viral vectors, purified from HEK 293 cells, using the 5-CD Gyrolab xPand system.


1:20 Chairperson’s RemarksHui Xiao, Senior Staff Scientist, Regeneron Pharmaceuticals, Inc.

1:25 Optimization and Application of Host Cell Protein (HCP) Analysis by Nano LC-MSGang Xiao, MSc, Senior Scientist, Process Development, Amgen, Inc.HCPs are process related impurities of biopharmaceuticals and application of MS to identify and quantify them is becoming more routine. This presentation describes optimization of HCP analysis by nano LC-MS using the NIST monoclonal antibody standard. The optimization is aimed at minimizing HCP loss from sample storage and handling, increasing HCP peptide recovery from the sample preparation, maximizing chance of HCP peptide detection, and reducing interfering from the biopharmaceutical proteins.

1:55 CO-PRESENTATION: Throughput Upstream Processing and Quality Analysis via Microfluidic Capillary ElectrophoresisJames Geiger, Field Application Scientist, Life Science Solutions, PerkinElmerSpeaker to be Announced


REGULATORY AND STANDARDS ISSUES (VIRTUAL SESSION)

2:40 Incorporating Mass Spectrometry into Biotherapeutic CMC Analytical StrategiesMatthew Maust, PhD, Investigator, GlaxoSmithKlineHistorically, immunoassays have been relied upon to characterize residual host cell proteins (HCPs) in biopharmaceutical drug substances. However, in recent years, due to the biochemical diversity found in HCPs, mass spectrometry has gained increasing acceptance as a tool for characterizing HCP profiles. As a relatively time consuming technique, it is important to understand how mass spectrometry-based assays fit most effectively within the whole analytical strategy for bioprocess development.

3:10 Best Practices for Identification and Quantitation of HCP Impurities in Biological Products Using Mass Spectrometry – Update from USP Host Cell Protein Expert PanelYing Zhang, PhD, Principal Scientist, Analytical Research and Development, Pfizer Inc.Mass spectrometry (MS) has become an increasingly common approach for identification and quantitation of host cell proteins (HCPs) in biotherapeutic products. Based on feedback from industry stakeholders, USP has formed an expert panel comprised of subject matter experts to write a new general chapter on best practices for identification and quantitation of HCPs by MS. Here we present an update on the Expert Panel’s progress and the chapter contents.





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6:30 Close of Day

TUESDAY, AUGUST 17


EMERGING METHODS AND TECHNOLOGIES

7:55 Chairperson’s RemarksKent Simmons, Senior Conference Producer, Cambridge Healthtech Institute

8:00 Establishment of a Walk-Up Fully Automated Ella Lab for CHO HCP AnalysisKathleen Van Manen-Brush, PhD, Investigator, Structure & Function Characterization, GlaxoSmithKlineCHO HCPs are processed-related impurities that are derived from the host cell expression system. Throughout process development HCPs are evaluated using ELISA. ELISAs are labor intensive with multiple manual steps with a timeframe of 7 hours. Ella is an automated “ELISA”-like technology that quantitates HCPs in 75 minutes. Both the Ella versus ELISA were comparable in CHO HCP trends, which resulted in development of a fully-automated CHO HCP analysis laboratory.

8:30 Compiling an HCP Knowledge RepositoryMichelle Busch, Scientist, Bioanalytics Characterization, SanofiHost cell proteins (HCPs) are process-related impurities that are monitored to ensure product purity, stability, efficacy, and safety. HCP levels were collected into a database from the analyses of hundreds of samples from the harvest and purification of biologics. Knowing the most abundant, problematic, and dynamic HCPs along with their physiochemical properties will help optimize upstream and downstream processes for a given protein product.

9:00 Improved Host Cell Protein Analysis in Monoclonal Antibody Products through ProteoMiner EnrichmentHui Xiao, Senior Staff Scientist, Regeneron Pharmaceuticals, Inc.This study reports a powerful strategy to identify HCPs in antibody drug substance by applying ProteoMiner enrichment with optimized conditions followed by shotgun proteomic analysis. Using this strategy, we observed that the low abundance HCPs were enriched up to 1000-fold. When applying this methodology to the study of HCPs in NIST monoclonal antibody (NISTmAb), more than 500 HCPs were confidently identified.

9:30 HCP Immunoassay Qualification: Review of Orthogonal MethodsEric Bishop, Vice President, Research and Development, Cygnus TechnologiesA broadly reactive and well qualified HCP ELISA is critical for monitoring purification process consistency and final drug substance purity. Here we present an overview of assay qualification steps and demonstrate how the orthogonal antibody affinity extraction and mass spectrometry methods assess ELISA Ab coverage to HCPs present in a given process and identify process-specific HCPs that may co-purify with a drug substance.


LC-MS IN HCP DETECTION AND CONTROL10:45 Host Cell Protein Profile Identification in a Cell Therapy Product Using LC/MS/MS TechniqueLei Wang, PhD, Associate Scientific Fellow, Analytical Development, Takeda PharmaceuticalsIn exploring Takeda cell-therapy pipeline, the retro-viral vector DS is generated by collecting culture media supernatant producing cells derived from a specific mouse cell line. Since no appropriate ELISA HCP kit is available, LC/MS-based shotgun proteomics is applied for HCP identifications during early phase. This presentation describes the HCP profiling procedure in DS and DP. It also includes a preliminary immunogenicity risk assessment based on in silico tool.





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11:15 Host Cell Protein Comparability Before and After Process Change by LC-MS Based Proteomics MethodTingting Jiang, PhD, Senior Scientist, Merck & Co., Inc.Host Cell Proteins (HCP) are process-related impurities that must be carefully considered during commercialization of biological products as they may impact safety and/or efficacy. Process changes in the upstream/downstream can potentially induce different HCP profiles. LC-MS based proteomics methods are an orthogonal approach to ELISA to identify/characterize individual HCP. LC-MS comparison of HCP process clearance and populations before and after process changes help ensure a robust commercial control strategy.

11:45 Toolkit for Profiling Low-Abundant Host Cell Proteins in BiotherapeuticsMidori Greenwood-Goodwin, PhD, Technical Development Scientist, Genentech, Inc.Low levels of host cell protein (HCP) impurities are difficult to remove completely and may impact drug efficacy or safety. Using a fast, robust 1D LC-MS/MS method for HCP identification, we can monitor HCPs present at low levels in multiple matrices. For specific HCPs, we leverage quantitative, high sensitivity analytical methods to support implementation of end-to-end mitigations, reducing overall HCP burden and total HCPs present in purified pools.

12:15 pm Sponsored Presentation (Opportunity Available)



CONTROL STRATEGIES1:55 Chairperson’s RemarksAbraham M. Lenhoff, PhD, Chair & AP Colburn Professor, Chemical & Biomolecular Engineering, University of Delaware

2:00 Doing the Right Things Right: HCP Control Strategy for BiopharmaceuticalsThomas Waerner, PhD, Associate Director & Project Manager, Analytical Development & Quality Control, Boehringer Ingelheim Pharma GmbH & Co. KG, GermanyThe control strategy to monitor residual HCP content in biotechnological products undergoes a steady evolution to ensure the best possible product quality and patient safety. With focus on HCPs derived from mammalian cells, this talk discusses different technologies for HCP detection considering the lifecycle status of the product (“doing the right thing”) and highlights relevant quality aspects for selected analytical methods (“doing the right thing right”).

2:30 Identification, Validation and Clearance of Highly Active Host Cell Proteins Responsible for Rapid Polysorbate Degradation in Formulated Drug ProductSisi Zhang, Lead Researcher, Regeneron Pharmaceuticals, Inc.PS80 degradation was observed in a monoclonal antibody (mAb) within 18 hours at 5 ºC with a low level of host cell proteins (HCPs) presented. This study focused on identification of the unique HCPs presented in this mAb that was responsible for the rapid degradation by applying a novel targeted HCP analysis method (Activity Based Protein Profiling (ABPP)). A twist of the ABPP method can facilitate the clearance of these HCPs.

3:00 Mechanisms of Persistence of CHO Host-Cell Proteins in mAb BioprocessingAbraham M. Lenhoff, PhD, Chair & AP Colburn Professor, Chemical & Biomolecular Engineering, University of DelawareIndividual CHO host-cell protein (HCP) levels may persist in mAb process streams despite the apparent effectiveness of separation processes, especially Protein A chromatography, at removing them. Several potential mechanisms have been explored for such persistence, such as HCP-mAb binding. This presentation will present results of recent such investigations and discuss possible mitigation approaches.

3:30 Control Strategy for Difficult to Remove HCPs in Biopharmaceutical DevelopmentSuli Liu, PhD, Senior Scientist, Analytical Development, BiogenA high-throughput and high sensitivity LC-MS based platform has been developed to monitor HCP clearance during process development. Two residual HCPs were identified as ‘difficult to remove’ during downstream purification process. Risk assessment was performed to understand the impact of the HCPs and to guide process development. An end-to-end approach from upstream to downstream was utilized to understand HCP removal capability to establish a robust process for biopharmaceutical manufacturing.



IN-PERSON INTERACTIVE DISCUSSION: Selection of Analytical Methods for Host Cell ProteinsHui Xiao, Senior Staff Scientist, Regeneron Pharmaceuticals, Inc.

5:45 Close of Host Cell Proteins Conference





8th Annual

Accelerating Analytical DevelopmentOptimizing the Speed and Efficiency of Key Analytical Steps in Biotherapeutic Development August 18-19

All Times EDT




AUTOMATION AND MINIATURIZATION7:55 Chairperson’s RemarksJanna Liptak, Principal Research Associate, Bioanalytics, Sanofi Genzyme

8:00 KEYNOTE PRESENTATION: Risk Assessment for Accelerated TimelinesRajiv M. Panwar, PhD, Director, CMC Operations, Disc MedicineCMC timelines are often aggressive and depend upon

CDMO’s platform processes and analytical methods. A thorough analysis and risk assessment of platform dependent product quality characteristics should be performed before finalizing the process for large scale antibody production. This presentation will cover our approach to risk assessment of platform dependent charge heterogeneity profile and how it enabled us to maintain our CMC timelines.

8:30 Enabling Technologies for Mass Spectrometric Characterization of BiologicsHarsha Gunawardena, PhD, Senior Scientist, Mass Spectrometry, Janssen Pharmaceutical Companies of Johnson & JohnsonThere is an emergent need for alternative technologies to support the characterization of molecules that are increasingly complex. We present several enabling mass spectrometry-based technologies that will dramatically increase the throughput in biologics R&D. We present the utility of microdroplets to perform real-time digestions, high-resolution ion mobility separations to unravel structural complexities, and multiplexing with isobaric labeling as a new paradigm for quantitation with phenomenal precision and unprecedented speeds.

9:00 Automation & Digitalization in High-Throughput Analytics for BioprocessRuchir Shah, Scientist, Process Development Analytics, Bristol-Myers Squibb Co.Bioprocess analytics have been challenged by the rapid growth of biomolecules. The application of assay automation tools and digital infrastructure allows high-throughput testing and rapid availability of analytical data for stakeholders. Our automation capabilities include the utility of robotic liquid handlers for sample extraction, preparation and/or pre-treatment for labor intensive workflows. Analysis, data piping from software to electronic notebooks real time visualization of results, trends, analytical methods and molecular information.

9:30 Large Molecules Get SLIM: Fast and Informative Large Molecule Characterization with High-Resolution Ion MobilityAndreas Krupke, PhD, Senior Product Manager, MOBILion SystemsHow can High-Resolution Ion Mobility Mass Spectrometry (HRIM-MS) address the challenges of large molecule characterization? This seminar will discuss the benefits of HRIM-MS in enhancing structural characterization of large molecule therapeutics, while increasing throughput

and reducing reliance on LC-based methods. Specific focus will be on how HRIM-MS provides more complete information on coeluting structural isomers, as well as and on emerging workflows.


10:40 Using Advanced Machine Learning Methods for Faster Development of AnalyticsMoritz von Stosch, PhD, Chief Innovation Officer, Datahow, SwitzerlandFaster development of analytics is rendered possible by advanced machine learning methods. The acceleration becomes possible by reducing the number of experiments required to develop the analytical methods. The number of experiments can be reduced because, e.g., the correlations in the data renders parts of the observations redundant or historic data can be exploited to develop the current analytical test. We will show results for advanced spectral data analysis.

11:10 Use of AI to Identify Sample Properties with SpectroscopyElvira Kadaub, Data Management Engineer, GenentechRaman and NIR spectroscopy are emerging methods for the characterization of biopharmaceutical therapeutics. Despite recent developments in analytical and spectroscopic methods, rapid establishment of accurate models for spectra processing remains a challenge due to statistical and biological uncertainties, time-consuming analysis. In that regard, we employed genetic algorithms to optimize Raman and NIR processing pipelines. This innovative approach demonstrates improvement in time, cost, versatility, and correlations across various categories of analytes.

11:40 Method Development for AutomationJanna Liptak, Principal Research Associate, Bioanalytics, Sanofi GenzymeAutomating sample preparation on robotic liquid handlers provides high-throughput capability and consistency during drug research and development. While some methods require minimal adjustments post-implementation, complex workflows often require continuous optimization and advancement. Presented here is the evolution of an automated sample preparation method for peptide mapping. What started as a semi-automated workflow has transformed into a fully automated process with optional front-end features and integrated analytical instrumentation.



CHARACTERIZATION SUPPORT FOR MATERIAL AND REAGENT SOURCING

1:25 Chairperson’s RemarksElena A. Smith, PhD, Deputy Director Quality Analytical Expert, Quality, Sanofi Pasteur





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1:30 Control Strategy Considerations for Ensuring Fit-for-Purpose MaterialsSusan E. Burke, PhD, Director Process Development, Materials Science, Amgen, Inc.The raw materials used in the manufacture of biopharmaceuticals are an important consideration for ensuring product quality. This presentation will describe key elements of our raw material control strategy including a framework for identifying important material attributes and a data analytics initiative for trending material performance. A case study will be presented to highlight how these tools are used to understand the relationship between material attributes and product quality attributes.

2:00 Seasonal Saga: Critical Reagents for Influenza VaccinesElena A. Smith, PhD, Deputy Director Quality Analytical Expert, Quality, Sanofi PasteurPotency assay for Influenza vaccines requires production of WHO reference materials (standard and strain-specific antibodies) each time when virus strains for vaccine composition is changed during the annual WHO strain selection process. Production of reagents is considered as a major factor delaying the vaccine supplies for the seasonal and potential pandemic influenza outbreaks. It necessitates tremendous efforts to manage critical reagents for the timely supply of vaccines each season.

2:30 Magnetic Resonance and Biopharmaceuticals, from R&D to Point of CareKate Holub, Business Development Manager, MRS Division, Bruker BiospinHigh-resolution NMR is a key technology that provides critical information about protein structure and dynamics in physiologically relevant conditions. Recent advances in acquisition and analysis enable us to see intact antibodies at natural abundance.1 Sensitive to changes in higher order structure, NMR is suited for similarity assessment of biologics and biosimilars.2 With intrinsically high information content NMR has the potential to reduce the number of techniques needed to characterize therapeutic drugs.





progress in recent years. Driven by this clinical success and the needs of commercialization, manufacturing strategies are progressing. Nevertheless, a standard manufacturing template for these therapies has not evolved and with novel modalities continuing to emerge we

may be in a post-template manufacturing landscape. CMC strategies must be developed to avoid manufacturing becoming a roadblock to therapeutic success.




6:00 Close of Day

THURSDAY, AUGUST 19


ASSAYS AND MODELS FOR LEAD SELECTION (VIRTUAL SESSION)

7:55 Chairperson’s RemarksKent Simmons, Senior Conference Producer, Cambridge Healthtech Institute

8:00 Sample Preparation for High-Throughput Lead SelectionPhylicia Dassardo-Joseph, Scientific Investigator, Biopharm Discovery, GlaxoSmithKlinePreviously in early biopharmaceutical drug discovery processes, only a finite selection of leads could be characterized due to limited material availability. In order to increase our screening capacity, automated sample preparation, assay miniaturization & improved data management strategies have been implemented. This has allowed us to create a data-rich environment enabling selection of high-quality leads. A high-level overview of this high-throughput workflow will be presented.

8:30 Implementing High-Throughput Tools for Lead Selection and Developability AnalysisAndrew K. Urick, PhD, Senior Scientist, AbbVieBiologics discovery projects require sensitive and high-throughput structural assays to select proteins with favorable developability profiles early in the discovery process. With pipelines frequently focusing on multiple distinct therapeutic modalities, these techniques must also be generally applicable. I will describe our strategy for implementing high-throughput analytics in our workflows with focus on sample consumption, throughput, and maximization of analytical diversity.






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9:30 Experimental and Computational Methods for Co-Optimizing Antibody Affinity and SpecificityEmily Makowski, Graduate Student, Tessier Lab, Pharmaceutical Sciences, University of MichiganTherapeutic antibody development requires co-optimization of biophysical properties that exhibit strict tradeoffs. This process remains challenging due to difficulties interpreting deep sequencing data. This presentation discusses advances in computational analysis of deep sequencing data, comparing several sequence-based feature extraction methods for analysis via machine learning models, ultimately achieving accurate predictions of analog binding strengths from binary sequencing labels and enabling the tunable co-optimization of antibody properties from high-throughput experimentation.

10:00 High Sensitivity Charge Variant Assessment of Biopharmaceuticals Using ZipChip Microchip ElectrophoresisJonathan Bones, Principal Investigator, Natl Institute for Bioprocessing Research & Training NIBRTThe use of the ZipChip device coupled to high resolution Orbitrap mass spectrometry is described for the characterization of charge variants of monoclonal antibodies under native conditions. The ZipChip provides excellent separation performance and facilitates impressive sensitivity based on operation under nanoelectrospray conditions, allowing for deep characterization from nanogram quantities of material.


IN-PERSON INTERACTIVE DISCUSSION: Analytical Strategies to Support Advancement of New TechnologiesElena A. Smith, PhD, Deputy Director Quality Analytical Expert, Quality, Sanofi Pasteur



CHALLENGES AND SOLUTIONS12:35 Chairperson’s RemarksIvie Aifuwa, PhD, Senior Scientist, Bristol-Myers Squibb Co.

12:40 wNMR Analytics of Biologics: IPC or Release Analytics Done in Seconds!Maximilian Hartl, PhD, Scientist & Lab Manager, Pharma Research & Early Development, Roche Diagnostics GmbH, GermanyTime-domain NMR (TD-NMR or wNMR) is a non-invasive and non-destructive method to analyze therapeutic biologics. Measurements can be done with small bench-top devices. On top of this: results are obtained within seconds. Thus, this technique is a perfect match for in-line analytics during manufacturing, especially continuous processing, or for real-time drug release. See here examples from our therapeutic protein and gen vector pipeline.

1:10 Accelerating Analytical Development: Utilizing Risk Assessments to Define When to Hit the Gas vs. the BrakesChristina Vessely, PhD, Senior Consultant, CMC Analytics & Formulation Development, Biologics Consulting Group, Inc.The early stage of development for a new biotech product is often hindered by lack of availability of materials and resources. This presentation is intended to enable the sponsor to utilize the data they have, in conjunction with risk assessments, to mitigate issues before they become major hurdles. The goal is to look beyond the numbers to get an understanding of what is really happening with the product.

1:40 Combining Unfolding Reversibility Studies and Molecular Dynamics Simulations to Select Aggregation-Resistant AntibodiesHristo Svilenov, PhD, Researcher, Technical University Munich, GermanyDuring this presentation, I will show how two approaches, the ReFOLD assay and modulated scanning fluorimetry (MSF), can be used to assess protein refoldability to select therapeutic antibody candidates that are resistant to aggregation during storage. Further, I will show how other biophysical methods and molecular dynamics (MD) simulations can support and explain the observations made with the ReFOLD assay and MSF.





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PROCESS ANALYTICS2:50 New Technologies for At-Line, On-Line and In-Line Process MonitoringIvie Aifuwa, PhD, Senior Scientist, Bristol-Myers Squibb Co.The iLine F is an on-line platform that provides near real-time measurements of VCC and viability. By employing a label-free approach, this platform utilizes the optical phenotype of expanding T cells and machine learning to classify cell viability and measure VCC. The iLine F can also interface with PCS to enable automated bioreactor operation, whereby enabling operators to inoculate the cell culture systems and walk away until harvest is required.

3:20 Establishing an Automated Raman-Based Chemometric Model Development PlatformDaniel R. Hill, Manufacturing Scientist, Global Process Analytics, BiogenAs the use of model-based applications supporting In-line Monitoring (ILM) and Real-Time Release Testing (RTRT) strategies increase, the need for efficient and robust model lifecycle management practices increases. Data integration and automated workflows provide a means for reducing long-term cost associated with multivariate models and enabling a more sustainable resource model. This talk will review effort at Biogen to design PAT systems and supporting workflows to enable automated modeling approaches.

3:50 Measuring the DNA Cargo of Viruses Using Nanofluidics and Machine LearningGeorgios Katsikis, PhD, Postdoctoral Associate, Koch Institute for Integrative Cancer Research, Massachusetts Institute of TechnologyRecombinant Adeno-Associated Viruses (rAAVs) deliver therapeutic DNA for gene therapy. However, rAAV manufacturing is imperfect, producing a small portion of recombinant viruses with the therapeutic gene. Here, we developed nanofluidic resonators for characterizing rAAV by measuring their mass. We juxtapose our approach with techniques ranging from ddPCR and Analytical Ultracentrifugation to Static Light Scattering. With our rAAV characterization approach, we aim to enable real-time quality control in continuous rAAV manufacturing.






Stability Stability & Impurities& ImpuritiesThe Stability & Impurities stream brings together experts in formulation, analytical sciences, and process scientists to share practical insights and case studies on rapid methods for analytical screening and development for traditional and novel formats, screening tools and strategies to manage contaminants and impurities. The two conferences will feature cutting-edge approaches for understanding and managing biologics product development, prediction and screening of stability, aggregations and excipient-related issues, product and process-related impurities, host cell proteins, lipases and enzymatic degradation, aggregates, degradants and more.

Detection, Characterization

& Control of Impurities in Biologics

Rapid Methods to Assess Quality &

Stability of Biologics

Conference Programs

AUGUST 16-17

AUGUST 18-19






9th Annual

Rapid Methods to Assess Quality & Stability of BiologicsImproving Prediction and Screening



MONDAY, AUGUST 16


IMPROVING IN SILICO PREDICTIONS9:55 Chairperson’s RemarksMark L. Brader, PhD, Research Fellow, Moderna Therapeutics, Inc.

10:00 KEYNOTE PRESENTATION: Expanding the Toolset of Models for Predicting Electrostatic Protein-Protein Interactions to Aid Candidate and Formulation SelectionChristopher J. Roberts, PhD, Professor, Chemical &

Biomolecular Engineering, University of DelawareDecisions regarding candidate selection for protein therapeutics need to incorporate many factors. One area that is challenging is the role of the surface charge distribution and the choice of solution conditions (e.g., pH, ionic strength). This presentation focuses on a coarse-grained molecular simulation approach that allows for rapid selection of amino acid mutations to aid candidate selection, with a focus on monoclonal antibodies as test cases.

10:30 In Silico Prediction of Physical and Chemical Stability of BiologicsNaresh Chennamsetty, Senior Principal Scientist, Bristol-Myers Squibb Co.In silico tools to rapidly assess physical and chemical liabilities of biologics such as aggregation, oxidation, deamidation will be discussed. Use of these tools for early risk assessment and to identify key critical quality attributes (CQAs) during manufacturing will be demonstrated. In addition, case studies will be discussed to obtain mechanistic understanding of the underlying cause of degradation and address unique stability challenges observed during development.

CHARACTERIZATION AND CONTROL STRATEGIES11:00 Analytical Challenges of a Novel Format Pegylated AntibodiesFeny Gunawan, Analytical Team Leader, Analytical Development & Quality Control, Genentech, A Member of the Roche GroupAdvancement in PEGylation technologies has provided new opportunities in drug discovery by enhancing clinically relevant properties of biological molecules. However, this new format presents new challenges for established analytical methods and controls, especially with respect to controlling charge variants. In this presentation, I will discuss how we overcome these challenges to design appropriate analytical controls to ensure purity and consistency for a novel PEGylated antibody.



12:50 Chairperson’s RemarksFeny Gunawan, Analytical Team Leader, Analytical Development & Quality Control, Genentech, A Member of the Roche Group

12:55 Control Strategy Considerations for Biophysical Attributes of Nanoparticle-Based ProductsMark L. Brader, PhD, Research Fellow, Moderna Therapeutics, Inc.The complexities of emerging nanoparticle-based delivery systems present specific challenges for the development and implementation of effective control strategies in early and late phase. A fundamental understanding of how biophysical attributes connect with product quality is needed to underpin effective analytical control. This talk will examine how physicochemical characteristics of nanoparticles may be considered together with challenges associated with leveraging instrumental methods for biophysical control.

1:25 Characterization of Adeno-Associated Virus (AAV)Sunny Zhou, PhD, Professor, Chemistry & Chemical Biology, Northeastern UniversityNew modalities present new challenges in their analysis. For example, due to their intrinsic structural complexity and complicated manufacture processes, viral capsids or particles (e.g., adeno associated virus, AAV) are markedly more heterogenous than well-established modalities. In this talk, both new methods and findings (e.g., PTMs) will be presented. Furthermore, I will discuss analytical artifacts, which are common yet under-appreciated, thereby often leading to erroneous interpretation and counterproductive approaches.



2:40 Implementation of High Throughput Analytical and Biophysical Tools to Support High Throughput Expression and PurificationChristopher Morgan, PhD, Assoc Dir, Dragonfly TherapeuticsDuring candidate lead selection, hundreds of molecules are screened for potency, binding and developability characteristics, such as hydrophobicity and thermal stability. As high-throughput expression/purification is implemented, analytical technology must adapt to increased number of samples, often with limited material availability. Here, data is presented showing implementation of high-throughput analytical technologies for CE, LC, and biophysical methods to support high-throughput screening processes to support lead selection.

3:10 Crystal Structure and Characterization of Human Heavy-Chain-Only Antibodies Reveals a Novel, Stable Dimeric Structure Analogous to mAbsCarl Mieczkowski, PhD, Associate Principal Scientist, Protein Sciences, Merck Research LabsIn this talk, we will discuss the novel crystal structure and characterization of human heavy-chain-only antibodies that reveals a novel, stable dimeric structure. Our findings indicate that human heavy-chain dimers can be secreted efficiently in the absence of light chains, may show good physicochemical properties and stability, are structurally similar to Fabs, their formation offer insights into their mechanism of formation, and may be amenable as a novel therapeutic modality.





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6:30 Close of Day

TUESDAY, AUGUST 17


IMPROVING DEVELOPABILITY & STABILITY7:55 Chairperson’s RemarksMark C. Julian, PhD, Scientist I, Biologics Drug Discovery, Biogen

8:00 FEATURED CO-PRESENTATION: Fast, Cost-Effective, and an Accurate Method for Measuring Protein Concentration: Comparison of Edelhoch Method with Other MethodsHaripada Maity, PhD, Director, Biologics Formulation Development, Jazz PharmaceuticalsPace et al. recommended an equation used to predict extinction coefficient of a protein. However, no antibody data was included in the development of this equation. The main objective of this study was to investigate how the predicted values are comparable to the experimentally determined values of mAbs measured by the Edelhoch method. A comprehensive analysis of the predicted and experimentally determined values by the Edelhoch method will be discussed.

8:30 FEATURED CO-PRESENTATION: Methods for Measuring Protein Concentration: Comparison of Edelhoch Method with Other MethodsDipanwita Batabyal, PhD, Process Development Scientist, Amgen, Inc.The objective of this study was to determine how the theoretical values of the extinction coefficient compares to the experimentally determined extinction coefficient for a large set of different biotherapeutic proteins measured by the Edelhoch method. Results clearly indicate that the Edelhoch method is highly reliable and shows a significant improvement in execution efficiency with reduction in cost and time when compared to the commonly used method, amino acid analysis

9:00 Testing and Improving Antibody Developability during Candidate Discovery and OptimizationMark C. Julian, PhD, Scientist I, Biologics Drug Discovery, BiogenThe ability to rapidly identify and reduce off-target binding early during candidate selection can help improve the odds of selecting lead antibodies with drug-like properties. I will discuss trends in antibody developability from our analysis of clinical and pipeline datasets across antibody modalities, and apply these learnings with a recent case study that utilizes predictive library design and high-throughput developability screening to improve specificity during optimization.

9:30 Talk Title to be AnnouncedDavid Sloan, PhD, Director, Applications, RedShiftBio


FORMULATION CONSIDERATIONS [VIRTUAL SESSION]

10:45 Co-Formulation Development: Balancing Challenges and Opportunities in Formulation and Process OptimizationAshlesha Raut, Senior Scientist, Biologics and Vaccine Formulation, Merck Research LabsCo-formulation dosing enables patient convenience and increased compliance due to simple one-vial image and single IV infusion, lowering the administration time. In addition to the combined therapeutic effect of two mAbs, co-formulation also offers advantage of efficient manufacturing processes. This presentation highlights key challenges and opportunities with case studies for co-formulation composition and process optimization supporting early stage development with line of sight to late stage.





9th Annual




11:15 Prediction of Formulation Parameters for High Concentration Antibodies Using High-Throughput TechniquesRajoshi Chaudhuri, PhD, Senior Scientist, Vaccine Production Lab, NIH NIAIDFormulations for antibodies at a high concentration are required to provide optimal conformational and colloidal stability. DOE methodology is often used to identify the pH, ionic strength and excipients, followed by real-time assessment of stability. This talk will discuss high throughput techniques to predict conditions for conformational stability and solubility and compare to real-time stability generated for multiple high concentration antibody formulation.”

11:45 Intelligent Formulation Development for Late-Stage High Concentration Biologics: Employing Previous Product Knowledge and Bridging Gaps CreativelySachin Dubey, PhD, Head of Formulation and Analytical Development, Glenmark PharmaceuticalsDeveloping robust formulation in a short development time is the need of the hour. Efficient use of prior knowledge and available product understanding are important considerations. An in-house case study will be presented, demonstrating a thorough data mining followed by a detailed evaluation of choice and role of excipients. The case study will highlight the selection of polysorbate concentration required for optimal product stability.

12:15 pm Sponsored Presentation (Opportunity Available)



BIOLOGIC STABILITY1:55 Chairperson’s RemarksMarc B. Taraban, PhD, Research Assistant Professor, Pharmaceutical Sciences, University of Maryland Baltimore

2:00 Benchtop NMR to Characterize Biologics StabilityMarc B. Taraban, PhD, Research Assistant Professor, Pharmaceutical Sciences, University of Maryland BaltimoreInteractions between water molecules and APIs in biologics make water protons a sensitive marker of the API structure and organization. As such, water proton NMR (wNMR) is advantageously used to quantitatively characterize the stability of biopharmaceutical drug products. Importantly, the analysis could be done quickly and noninvasively, in the original drug product container using low-cost wide-bore compact benchtop NMR spectrometers.

2:30 Evaluation of Chelators in Mitigating Polysorbate 80 Degradation in Biologic FormulationsAnvay Ukidve, PhD, Scientist, Formulation and Process Development, SanofiMetal ion impurities are responsible for the chemical degradation of formulation components such as the polysorbates and active protein molecule. The use of metal ion chelators, like EDTA and DTPA, to mitigate chemical degradation is a well-established strategy to tackle this problem. In this work, we assessed the impact of effectiveness of different types and different levels of chelators in mitigating Polysorbate 80 degradation.

3:00 Accelerated Strategies to Assess Interfacial Stability of BiopharmaceuticalsDanny K. Chou, PharmD, PhD, President, Biopharmaceutical Characterization and Formulation Development, Compassion BioSolution, LLCIn this talk, I will discuss the latest developments in rational strategies to evaluate the effects of surface-mediated stress on antibody instability. The focus will be on early detection and mechanistic understanding that will enable the creation of an effective control strategy in the industrial setting.

3:30 Novel Approach for Subvisible Particle Characterization of BiopharmaceuticalsSravan Penchala, PhD, Senior Scientist, Biologics Drug Product Development, Janssen Pharmaceuticals, Inc.Particle characterization of biologics is challenging especially for investigational drug products that are prone to particle formation during their development, manufacturing, and administration. To address some of the frequently observed challenges during particle characterization of intravenous-admixture compatibility studies, we are proposing a novel approach to characterize subvisible particles using existing tools such as light obscuration and flow imaging microscopy.



IN-PERSON INTERACTIVE DISCUSSION: Advances in Process Analytical Technology (PAT) for Therapeutic Protein and Cell/Gene Therapy Development and ManufacturingDanny K. Chou, PharmD, PhD, President, Biopharmaceutical Characterization and Formulation Development, Compassion BioSolution, LLC• What is the role of PAT in the biopharmaceutical industry? How does this

role differ between protein pharmaceutical development/manufacturing and Advanced Medicinal Products like gene therapy and cell therapy?

• What are some of the critical quality attributes that can be monitored by real-time methods and what the key gaps that remain unfulfilled?

• What can we do to make PAT more efficient and easy to implement in the real world?

5:45 Close of Rapid Methods to Assess Quality & Stability of Biologics Conference





4th AnnualDetection, Characterization and Control of Impurities in BiologicsContaminants and Impurities, Case Studies and New Technologies and Strategies August 18-19

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PROCESS-RELATED IMPURITIES7:55 Talk Title to be AnnouncedMichael Dolan, Staff Engineer, Process Development US, Takeda Pharmaceuticals

8:00 KEYNOTE PRESENTATION: Systematic and Deep Characterization of Host Cell Proteins (HCPs)Sunny Zhou, PhD, Professor, Chemistry & Chemical Biology, Northeastern UniversityMichael Dolan, Staff Engineer, Process Development US, Takeda PharmaceuticalsHost Cell Proteins (HCPs) in biotherapeutics have the potential to affect product quality, and immunogenicity in patients. In this talk, we will discuss the regulatory requirements for HCPs, profiling parameters, and

characterization tools and methodologies.

9:00 Sensitive, Rapid, Robust, and Reproducible Workflow for Host Cell Protein Profiling in Biopharmaceutical Process DevelopmentJiao Ma, PhD, Senior Scientist, AstraZenecaThere is a growing industry and regulatory need to detect host cell proteins in the production of protein biopharmaceuticals, as certain HCPs can impact product stability, safely and efficacy, even at low levels. We developed an LC-MS/MS based HCP profiling workflow to detect and quantify HCPs in high-throughput manner. The workflow can be readily implemented to guide biopharmaceutical process development and enable better risk assessment of HCPs in drug substances.

9:30 Combining Deep Learning & Imaging Microscopy to Analyze and Monitor BiologicsChris Calderon, PhD, President, Ursa AnalyticsCompendial methods to analyze subvisible particles in biologics often encounter difficulties when applied to proteinaceous drugs. This presentation discusses how to combine AI, computational statistics and high-throughput microscopy to characterize particles (e.g., protein aggregates) in biologics. By analyzing morphological and textural features particles in images, this method delivers quantitative, actionable information for formulation development, container qualification, and fill-finish quality control. Four case studies are discussed.


10:40 Non-clinical and Clinical Experience for Endotoxin and Beta Glucan in Intravitreal Administration for Ocular TherapeuticSara Parker, Senior Manager, Process Development, Genentech, A Member of the Roche Group



IMPURITIES & STABILITY1:25 Chairperson’s RemarksDanny K. Chou, PharmD, PhD, President, Biopharmaceutical Characterization and Formulation Development, Compassion BioSolution, LLC

1:30 FEATURED PRESENTATION: In-Line Monitoring of Surfactant Clearance in Viral Vaccine Downstream ProcessingMarina Kirkitadze, PhD, Head Bioprocess Support & PAT Platform, Analytical Sciences, Sanofi Pasteur

We examined suitability of in-line infrared (IR) measurements to monitor, in real-time, surfactant conc. in the viral vaccine drug substance. Off-line sample data were found to be in good agreement with surfactant conc. values obtained by HPLC. The results of a small-scale in-line study demonstrated suitability of an in-line IR measurement to monitor surfactant concentration in the viral vaccine drug substance between exchanges 8-15 of a 50kDa TFF process.

2:00 Analytical Control Strategy of Impurities during Manufacturing of Therapeutic ProteinsKevin Zen, PhD, Executive Director, Analytical Development, Chemistry, Manufacturing and Controls, AnaptysBio Inc.The therapeutic proteins are manufactured through upstream/downstream process, fill/finish, and delivered to patients. Impurities in therapeutic protein drugs can originate from raw material, bioprocess, product itself, or dosing regimen. This presentation will highlight potential impurities of therapeutic proteins and share the best practice of analytical procedures and characterization technologies to monitor and control impurities. The impurity comments/questions from health authority after IND/IMPD is submitted will be exemplified as case studies.

2:30 Viral Clearance Prediction for Downstream Process Development and CharacterizationDavid Cetlin, Senior Director, MockV Products, Cygnus TechnologiesVirus Like Particles can be used to economically predict viral clearance outcomes during process development and characterization in a BSL-1 setting. This presentation will first review the physicochemical characteristics of a non-infectious Minute Virus of Mice particle and a CHO-endogenous Retrovirus Like Particle. Then, application data will be shown from a series of comparative spiking studies including an AEX DOE study, a HTS resin selection study, and an AAV process study.









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6:00 Close of Day

THURSDAY, AUGUST 19


PRODUCT-RELATED IMPURITIES7:55 Chairperson’s RemarksSanket Patke, PhD, Associate Director, Sanofi

8:00 A Systematic Approach to Drug Product Process Development and Technology Transfer to Commercial Manufacturing SiteSanket Patke, PhD, Associate Director, SanofiIn this presentation, we will discuss an approach to perform drug product process development studies to de-risk DP manufacture at commercial manufacturing site. We also discuss a QbD approach for technology transfer.

8:30 Opportunities and Challenges in Real-Time Monitoring of Protein Aggregation during Biopharmaceutical Development and ManufacturingDanny K. Chou, PharmD, PhD, President, Biopharmaceutical Characterization and Formulation Development, Compassion BioSolution, LLCReal-time analytical techniques provide an opportunity for more effective monitoring and control of protein aggregation in bioprocessing as well as a better understanding of the mechanisms of protein aggregation. The objective of this presentation is to share these opportunities as well as challenges in the implementation of novel technologies that can aid in the detection of protein aggregates in real-time.


9:30 Characterizing Medium Hydration Using Process Analytical TechnologiesKyle Devenney, MS, Scientist, MerckVariability throughout bioprocess manufacturing is magnified by impurities introduced by the raw materials and medium mixing protocols. Raman spectrophotometry and focused beam reflectance measurement (FBRM) allow us to capture the intricate media chemistry that occurs due to the changes introduced by medium raw material impurity or mixing parameters. In this presentation, we will share our strategy and case study on using analytical tools in characterization of medium preparation.



IN-PERSON INTERACTIVE DISCUSSION: Impurities in BiologicsSunny Zhou, PhD, Professor, Chemistry & Chemical Biology, Northeastern University• What are the emerging issues and problems in your area?• What specific individual HCPs have gained a deeper understanding in the

past years?• What specific individual HCPs are still lacking in understanding but should

be prioritized?• What are the new features for new modalities, such as gene therapy?







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PARTICLES AND SURFACTANT-RELATED IMPURITIES (VIRTUAL SESSION)

12:35 Chairperson’s RemarksJiao Ma, PhD, Senior Scientist, AstraZeneca

12:40 Role of Interfaces in Particulate Formation of BiologicsItzel Condado Morales, PhD, Biochemical Engineering Laboratory, Institute for Chemical and Bioengineering, ETH ZurichThe aggregation of biotherapeutics is an undesirable phenomenon for various reasons, including reduced efficacy as well as immunogenicity. Nevertheless, the aggregation process is often difficult to understand and more importantly, to predict for newly developed molecules. Here we present a method for studying the surface-mediated stress that leads to aggregation, in a very controlled manner. The method was applied to a library of antibodies to select the best candidates.

1:10 Characterizing Surfactant/Preservative Interactions Using Small-Angle ScatteringPeter H. Gilbert, Postdoctoral Researcher, Center for Neutron Research, National Institute of Standards and Technology (NIST)Polysorbate 80 (PS80), a nonionic surfacatant used in pharmaceutical formulation, is known to be incompatible with m-cresol, an antimicrobial agent. This incompatibility results in increased turbidity caused by unsteady micelle aggregation or coalescence. PS80/m-cresol solution phase instability progesses through multiple growth stages that can be explained using a single kinetic model. We use small-angle neutron scattering (SANS) to reveal the necessary conditions for aggregation, the coalescence mechanism and aggregate structure.

1:40 Formulation Strategies to Minimize Particles Resulting from Polysorbate DegradationNidhi Doshi, Scientist, Late Stage Pharmaceutical Development, Genentech Inc.Free fatty acid particles associated with enzymatic polysorbate degradation in liquid drug products is of increasing concern to the biopharmaceutical industry. There are currently several gaps in the industry’s understanding of how to mitigate this issue. The talk will focus on formulation related considerations and risks associated with polysorbate degradation as well as strategies on how to manage polysorbate degradation and reduce particle risk.








Gene Gene TherapyTherapyThe Gene Therapy stream features two back-to-back conferences focusing on the critical challenges facing the analysis, characterization, quality control and manufacture of viral vector-based gene therapies. Topics include product and process characterization, analytics and quality profiling, comparability, process development, scale-up and commercial manufacturing.

Gene Therapy Manufacturing

Gene Therapy CMC and Analytics

Conference Programs

AUGUST 16-17

AUGUST 18-19






5th Annual

Gene Therapy CMC and AnalyticsAdvancing Product Quality and Process Understanding August 16-17

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MONDAY, AUGUST 16


CMC STRATEGIES FOR GENE THERAPIES9:55 Chairperson’s RemarksJames Warren, PhD, Vice President, Pharmaceutical Development, Ultragenyx Pharmaceutical

10:00 KEYNOTE PRESENTATION: Current Challenges in Gene Therapy Technical DevelopmentMark Galbraith, PhD, Former Head, Quality Control and Analytical Sciences, Spark Therapeutics

Implementation of a manufacturing process that assures a predefined quality of product is a critical requirement for the licensing and marketing of every CGT product. This presentation will discuss the current challenges in gene therapy technical development to ensure safe, well-characterized products.

10:30 FEATURED PRESENTATION: Potential Challenges Throughout the Development ProcessAndrew W. Harmon, PhD, Biologist, CMC Reviewer, Division of Cellular and Gene Therapies, Office of

Tissues and Advanced Therapies, FDA CBERThis presentation will discuss common challenges that may arise during development of Gene Therapy products. Topics will include the importance of anticipating the next steps in product development to ensure that early activities, including product characterization and assay development, can support potentially rapid clinical study progression. Regulatory mechanisms available to facilitate development of promising Gene Therapy products will also be highlighted.

11:00 Analytical Strategies for Recombinant AAV Characterization and Product ReleaseZhu Zhen Pirot, PhD, Vice President, Translational Science, Kriya TherapeuticsManufacture scale up and analytics are key challenges in AAV gene therapy development. Establishment of clear analytical strategies aligning with process & manufacture development from early translational development is critical for speed-to-clinical development. The presentation will discuss our integrated strategies and some key analytical development.

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11:30 Vector Safety Assessment in Cell and Gene TherapyWei Wang, PhD, Director, Business Development, GeneWerkGeneWerk specializes in vector safety and integration site analysis for gene- and cell- therapy. We also offer a wide variety of custom-tailored services around safety and efficacy, such as analyses for gene-editing on/off-targets, TCR/BCR immune repertoire, or AAV impurities. Our goal is to assist clients in moving towards safer therapies.


CHARACTERIZATION AND POTENCY ASSAYS12:50 Chairperson’s RemarksMark Galbraith, PhD, Former Head, Quality Control and Analytical Sciences, Spark Therapeutics

12:55 Stability-Indicating Assays for AAV-Based VectorsMarina S. Feschenko, PhD, Director, Gene Therapy, BiogenAAV-based vectors used in Gene Therapy are new and complex modalities both in terms of structure and function. The mechanism of action of viral vectors is multifaceted, which requires matrixed approach for assessment of biological activity. This presentation will focus on stability-indicating methods for AAV-based vectors and will discuss challenges and opportunities for structure-activity relationship studies.

1:25 Potency Assay Strategy for AAV Gene TherapiesAisleen McColl-Carboni, PhD, Director, Analytical Development, Homology Medicines, Inc.AAV gene therapy products have complex mechanisms of action that pose unique challenges to potency assay development. Determining the true biological activity often requires multiple assays (i.e. a matrix approach), and the strategy for implementing these assays may evolve through the product lifecycle. This presentation will discuss phase-appropriate development and qualification of different in vitro potency assays.

1:55 TEM Analytics to Check Purity, Integrity, and Formulation Changes in the Production of Viral Vectors and VaccinesQiong Wu, PhD, Senior scientist, Electron Microscopy Services, Vironova BioAnalytics ABVironova revolutionizes transmission electron microscopy (TEM) for nanoparticle characterization in the fields of gene therapy, vaccines, and drug delivery based on image analysis. The high-resolution images can provide essential information of morphology, packaging, integrity, and purity of the sample. Our in-house image analysis software and GMP-certified laboratory provide TEM services that answer questions which arise during the production process, helping in the decision-making in viral vector development.


ANALYTICAL STRATEGIES (VIRTUAL SESSION)2:40 Characterization of DNA Starting MaterialsLawrence C. Thompson, PhD, Senior Principal Scientist, Analytical R&D, Pfizer Inc.The release testing package for plasmid DNA starting materials is more or less a platform set of assays while the amount (if any) of characterization testing is much less defined. This presentation will discuss 4 different examples of nucleic characterization tools and how they can be used to enhance the release methods and probe the quality of the starting material a little deeper than previously considered.





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3:10 Advanced Analytics for Virus-Based Gene TherapiesShibani Mitra-Kaushik, PhD, Director & Head, Bioassay & Molecular Analytical Development, Genomic Medicine Unit, SanofiVirus-based gene therapies require complex analytical methods to assess key quality attributes during process development, GMP release and stability monitoring of clinical grade product. This presentation will discuss phase appropriate paradigms for analytical method development, qualification, and implementation into gene therapy programs as they transition from research into clinical stages. Some challenges with material and time limitations and proposed resolution strategies using platform-based methods will be explored as well.









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TUESDAY, AUGUST 17


ANALYTICAL STRATEGIES FOR GENE THERAPIES7:55 Chairperson’s RemarksAdriana Kita, PhD, Associate Director, Analytical Development, Ultragenyx

8:00 Novel Methods for AAV Product CharacterisationTony Bou Kheir, PhD, Lead Technical Scientist, Cell and Gene Therapy CatapultAs the gene therapy field continues to expand and companies increase the number of product batches manufactured per year, the burden on quality control for product release increases significantly. The presentation will showcase CGT Catapult’s advanced analytical platform for AAV product characterisation, which includes assay development for improving precision, assay automation and in-line product characterisation for closed processes.

8:30 Implementation Strategy of a Next-Generation Sequencing WorkflowJarrod Dean, Associate Director, Genomic Medicine Unit, SanofiThis presentation will include a case study detailing the validation of an NGS analysis pipeline for GMP program support, use of Agile methodology to track project progression and deliverables; and a proposed strategy to support the transfer of an NGS analysis pipeline from a development to production environment

9:00 Analytical Method for Determination of Expression of a Secondary Element in the Transgene Cassette by RT-PCRCarlos E. Peredo, PhD, R&D Associate Fellow, Cell and Gene Therapy Platform, GlaxoSmithKlineT cells transduced with self-inactivating lentiviral vectors that co-express affinity-enhanced cancer antigen T cell receptors (TCRs) and T cell function modulators may demonstrate antitumor efficacy superior to TCRs alone. This work describes an analytical method which demonstrates the expression of transgenic T cell function modulators distinctly from their endogenous counterparts for characterization of lentiviral vectors and transduced T cell products.

9:30 High Throughput Characterization of Antibody, AAV and Cell Therapy Aggregates Using the AuraBernardo Cordovez, PhD, Chief Science Officer and Founder, Halo LabsIn all biological products, distinguishing aggregated API from other particle types matters for understanding the root cause of instability. Until now, subvisible particle characterization methods have been unreliable, slow, and difficult to use across many workflows. Introducing the Aura, a 96-well, low-volume, high throughput aggregate and particle imaging system can rapidly size, count, and characterize biological particles and identify them as proteins, non-proteins, cellular aggregates, or other types of molecules.





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ANALYTICAL STRATEGIES FOR GENE THERAPIES10:40 Chairperson’s RemarksWei-Chiang Chen, PhD, Associate Director, Sanofi

10:45 Characterization of AAV Genome and Co-Packaged DNA Impurities by Next-Generation SequencingWei Zhang, Associate Director, Analytical Development, UltragenyxFor AAV gene therapy products, partial genomes and encapsidated residual DNAs impurities pose safety risk and compromise product efficacy. Their levels need to be monitored and controlled during product development. Next generation sequencing offers orthogonal approaches to traditional methods in characterizing such impurities, providing insights on genome integrity, DNA impurities levels, and size.

11:15 Improved qPCR Methods for AAV Genome Titer and Residual DNA QuantificationYu Wang, PhD, Senior Scientist, Analytical Development, BiogenqPCR method is widely used to quantify genome titer and residual DNA in AAV gene therapy products. However, the standard method requires complicated sample treatment and is known to be variable. In this presentation, we will introduce a new method with optimization of AAV sample preparation and choice of qPCR standard, which not only simplify the method, shorten the assay time, but also improve overall assay performance.

11:45 AAV Capsid Quantification Methods and Its Relevance to Other Gene Therapy Product Quality AttributesAdriana Kita, PhD, Associate Director, Analytical Development, UltragenyxCapsid titer can be measured using a variety of analytical techniques and is important for both early and late stage process development. Here, we compare methods for measuring AAV capsid titer and evaluate these measurements against other product quality attributes including genome titer and empty/full capsid ratios.

12:15 pm Process Development Strategy of Adeno-Associated Viral VectorKenneth Warrington, Senior BD Director, Strategy & Business Development, GenScript ProBioA growing number of gene therapy products are currently in preclinical or clinical trials to explore the potential in the treatment of multiple diseases. Among gene therapy vehicles, adeno-associated viral vector (AAV) is one of the most actively investigated deliver vectors with superior safety. GenScript ProBio helps solve the challenge of scalable and stable manufacturing for AAV from preclinical through IND application to clinical phases, bring excellent solutions to gene therapy.

12:30 Standardizing rAAV Production for Gene TherapyNeal Goodwin, PhD, Chief Scientific Officer, TeknovaTeknova is developing an optimized ion exchange (IEX) chromatography algorithm to purify full rAAV viral vectors that account for vector diversity. We rapidly assessed multiple parameters and created customized protocols and reagents for downstream processing. To date, we have used the formulated algorithm to develop optimal IEX protocols and ready RUO or GMP reagents for purifying multiple serotypes and payloads.



ADVANCED ANALYTICAL STRATEGIES1:55 Chairpersons’s RemarksWei-Chiang Chen, PhD, Associate Director, Sanofi

2:00 Advanced Analytics for AAV CharacterizationSantoshkumar L. Khatwani, PhD, Director, Analytical Development, Sangamo TherapeuticsThis talk will focus on advanced analytics of AAV with respect to critical quality attributes - vg titer, empty full AAV, aggregation, MS. In addition, this talk will show examples of orthogonal approaches to switch from traditional to advanced analytics for AAV.

2:30 Expedite Gene Therapy Development by Advanced Mass Spectrometry CharacterizationVictor Chen, Principal Scientist, RegenxbioGene therapy products have demonstrated a great potential to treat devastating diseases and are being extensively evaluated in clinical trials for many disease indications. The structural and biological properties of these products are complex and yet to be fully realized. Advanced mass spectrometry applications are developed to assess AAV vector quality attributes. Case studies of mass spectrometry applications in expediting gene therapeutic development will be presented.

A-GENE, QbD, MAM3:00 Update on A-Gene: Best Practices for the Manufacture of AAV VectorJosephine Lembong, PhD, Manager, Science and Industry Affairs, Alliance for Regenerative MedicineThe Cell & Gene Therapy industry is rapidly growing with more commercial product approvals anticipated each year. However, the lack of standardization and best practices around CMC programs creates hurdles to streamlined, cost-effective manufacture. Borrowing from the ‘A-Mab’ model in the monoclonal antibody field, the Alliance for Regenerative Medicine has produced a similar document titled A-Gene, a case study-based approach to integrating Quality by Design principles in gene therapy manufacturing.





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3:30 QbD for Gene TherapiesJessie Sun, PhD, Director, Ultragenyx Pharmaceutical, Inc.


4:45 Implementation and Adaptation of Multi-Attribute Mass Spectrometry for Adeno-Associated Virus (AAV) Vector CharacterizationJoshua Powers, PhD, Post-Doctoral Associate, NIST Biomolecular Measurement DivisionGene therapies possess substantial potential to be revolutionary curative treatments, but their broad implementation requires further progress in biomolecular understanding. High-resolution analytical approaches must be adopted to fully characterize these molecularly complex treatments and monitor the final drug product. A multi-attribute mass spectrometry method was developed to monitor AAV vector quality attributes and detect new peaks detection, which should be widely applicable to other vector-based modalities.


CO-PRESENTATION: IN-PERSON INTERACTIVE DISCUSSION: Gene Therapy AnalyticsWei-Chiang Chen, PhD, Associate Director, SanofiVictor Chen, Principal Scientist, Regenxbio

5:45 Close of Gene Therapy CMC and Analytics Conference





6th Annual

Gene Therapy ManufacturingEnsuring the Clinical and Commercial Supply of Gene Therapies August 18-19

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ADVANCING GENE THERAPY MANUFACTURING7:55 Chairperson’s RemarksCarlos A Benitez, MSEM, Scientist, Purification Process Development, Genomic Medicine Unit, Sanofi

8:00 KEYNOTE PRESENTATION: Application of a Commercial Scale Producer Cell Line Platform to Enable Robust, Efficient CMC and Clinical Development of rAAV Gene Therapy ProductsJames Warren, PhD, Vice President, Pharmaceutical

Development, Ultragenyx PharmaceuticalUGT has established and optimized the HELA Producer Cell Line system, directly scalable to 2000L, and has applied this platform across multiple clinical development candidates. This platform enables a rapid CMC development strategy, through implementation of high-throughput centers of excellence, representative lab-scale models, and a state-of-the-art pilot plant to streamline and standardize the development and technology transfer of preclinical and clinical candidates to external manufacturing partners.

8:30 Challenges in Upstream Process Intensification for AAV ManufacturingMayur Jain, PhD, Group Leader, Upstream Process Development, Asklepios BioPharmaceuticalRecombinant adeno-associated viruses (rVVV) is small nonenveloped virus widely used as a gene transfer vehicle for in-vivo gene therapies. To date, generating vector quantities sufficient to meet clinical demand is still a hurdle when using the current production systems. AskBio has developed an intensified manufacturing process platform focusing on increasing cell densities and higher product rAAV yields.

9:00 How to Develop and Leverage an Integrated Manufacturing Process and Platform to Increase Speed to ClinicMichael Mercaldi, PhD, Senior Director, Downstream Process Development, Homology Medicines, Inc.Increasing the speed at which gene therapies can be manufactured and introduced into the clinic is critical to realizing their potential. To successfully do this, the development of a “plug and play” process and platform, created by leveraging platform knowledge, is crucial. By taking this approach, an organization can help to rapidly and effectively build out a robust gene therapy pipeline.

9:30 Optimizing Transfection Process in Suspension Cells to Reduce Dramatically the Cost of Goods of AAV Gene TherapyMatthias Hebben, PhD, Vice President, Technology Development, LogicBio TherapeuticsAAV-based gene therapy products have demonstrated promising results in clinical trials,but their manufacturing costs are so high that these treatments may not be affordable for most of the patients. To increase AAV vector yields, LogicBio optimized the plasmids used for suspension

HEK293 cell transfection. When combined with a novel transfection reagent, the vector titers were increased by 20 to 25 times, resulting in a significant drop of the cost of goods.


OPTIMIZING PROCESS DEVELOPMENT11:10 Optimizing Viral Vector Process DevelopmentStephen Soltys, PhD, Vice President, Process Development, Kriya TherapeuticsOur cGMP production suites and single-use systems will allow the production of multiple products simultaneously at up to 3,000-liter bioreactor scale. We are developing reliable and robust production systems that can deliver higher amounts of AAV product per batch.

11:40 Academic Vector Production for Early Phase Clinical Trials: Challenges and BottlenecksJohannes C.M. Van Der Loo, PhD, Director Clinical Vector Core, Perelman Center for Cellular & Molecular Therapeutics, Children’s Hospital of PhiladelphiaThe academic-based Clinical Vector Core (CVC) at the Children’s Hospital of Philadelphia has a 17-year track record in pre-clinical and clinical grade viral vector manufacturing for early phase clinical trials. The growth of the field has created unique challenges driven by the need for more and larger products, progression of studies to later phases of development, increased involvement of investors and industry, and increase in international collaborations and regulatory scrutiny.

12:10 pm LUNCHEON PRESENATION: Optimized, Efficient, and Scalable Transient Transfection for High Titer AAV and LV Generation.Leisha Kopp, Applications Scientist, Mirus BioRecombinant adeno-associated virus (AAV) and lentivirus (LV) are key components in many gene and cell therapies designed to treat a vast array of human diseases. We will discuss optimization strategies for maximizing AAV and LV titers through transient transfection of adherent and suspension HEK 293 cells for both small- and large-scale processes. Attendees will also learn how VirusGEN® GMP AAV and LV Kits support gene and cell therapy researchers from R&D through commercial manufacturing.


OPTIMIZING PROCESS DEVELOPMENT1:25 Chairperson’s RemarksCarlos A Benitez, MSEM, Scientist, Purification Process Development, Genomic Medicine Unit, Sanofi





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1:30 Development of a High-Yielding AAV Production ProcessMatthew Roach, AAV Process Development Team Leader, Precision BioSciencesThe use of adeno-associated virus as a vector for gene therapies continues to grow. Upstream production yields have lagged the clinical demand, and production remains a significant barrier to wide adoption. This talk will detail our journey through the development of a high-yielding AAV production process and the learnings we had along the way.

2:00 Optimization of Anion Exchange Chromatography to Enrich Full rAAV particlesChao Huang, PhD, Associate Director, Pharmaceutical Development, Ultragenyx PharmaceuticalGene therapy has an increased demand on full rAAV particles by high clinical doses. AEX is commonly used to separate empty and full particles by exploiting the difference in surface charges. Here we report several improvements to an existing AEX method to enrich full rAAV ratio. High throughput screening system and PAT tools were utilized to enable rapid process development.

2:30 Label-Free, Standard-Free, Hassle-Free AAV Empty/Full and Titer Data on StunnerRoss Walton, PhD, Senior Application Scientist, Analytics, Unchained LabsAAV titer, empty/full ratio, and aggregation state are critical readouts to any AAV production process. Stunner’s dye-free, label-free, and standard-free AAV quant delivers it all using just 2 µL of sample, in less than a minute - way faster than AUC, ELISA and ddPCR. Learn how Stunner’s hassle-free AAV characterization can help optimize your process.









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THURSDAY, AUGUST 19


OPTIMIZING PROCESS DEVELOPMENT7:55 Chairperson’s RemarksMeisam Bakhshayeshi, PhD, Director, Process Development, Tessera Therapeutics

8:00 Challenges in Lentiviral Vector Upstream Process: Engineering Cells and VectorsAna Sofia Coroadinha, PhD, Lab Head, Health & Pharma Division, Animal Cell Technology Unit Cell Line Development and Molecular Biotechnology Lab, IBETThe use of lentiviral vectors in cell therapies require the manufacture at large scale of high quality material to enable efficient cell transduction. This work discuss the main challenges lentiviral vector cell therapies face and present strategies and novel technologies to be adopted to enable effective manufacture as well as cell transduction. of cells.

8:30 Transitioning to a Suspension Platform: Trials and TribulationsBryan A. Piras, PhD, Lead Scientist, AAV Vectors, St. Jude Children’s Research HospitalLike many in the industry, our group has recently made the transition from adherent cells to suspension cells for the production of AAV. This talk will focus on the major challenges we have faced during this transition and how we have overcome them, with a focus on product yields and functionality, host-cell impurities, and the impact of specific cell lines.


9:30 Optimizing Process DevelopmentJacob M. Smith, PhD, Senior Director, Process Development, Asklepios BioPharmaceuticalRecombinant adeno-associated virus (rAAV) has emerged as a dominant gene-delivery vector of choice. Despite numerous advances in the clinic, the efficiency and cost of producing rAAV drug product to meet a rapidly growing industry has significant room for improvement. AskBio has developed a model for advancing multiple clinical programs, with an





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emphasis on establishing early benchmarks in manufacturing feasibility, while focusing on continuous process development and improvements to large-scale manufacturing.

10:00 Measuring AAV Quality Attributes with SEC-UV-MALS-dRIJohn Champagne, PhD, Senior Application Scientist/NE Regional Manager, Wyatt TechnologyAdeno-associated virus (AAV) is an attractive delivery vehicle in gene therapy attributed to its mild immune response and ability to deliver its genome into a range of host cells. This presentation will discuss a method to measure the following three important AAV quality attributes (QAs): 1) total number of viral capsid particles; 2) relative capsid content (e.g., ratio of empty and full capsids); and 3) percentage of monomer or aggregates.

10:30 Upstream Process Development and Characterization for Late Phase AAV Gene Therapy ProductsJessie Sun, PhD, Director, Ultragenyx Pharmaceutical, Inc.During the late stage development, the scale down model (SDM) becomes a critical tool for process characterization (PC). In this presentation, strategy for SDM development, qualification, and process characterization will be shared for HEK293 transient transfection system. In addition to monitoring cell growth performance, product titer and quality, other atline/offline analytical tools were employed to further characterize the transfection complex.


IN-PERSON INTERACTIVE DISCUSSION: Future Challenges in Gene Therapy ManufacturingMeisam Bakhshayeshi, PhD, Director, Process Development, Tessera Therapeutics

11:30 CO-PRESENTATION: LUNCHEON PRESENATION: Accommodating Adherence and Suspension Processes in a Single Intensified and Scalable PlatformDavid Moccia, Business Development, Univercells TechnologiesEffie Huang, Senior Application Specialist, Univercells TechnologiesThe continuously growing gene therapy market has revealed several challenges in viral vector manufacturing. The highly variable gene therapy capacity demand with dose sizes reaching up to 1E20 vgs exposes an

urgent need for flexible and scalable solutions. Traditionally it has been hard to find such technologies often resulting in process scale-out for high-demand applications. But what if a dual platform could enable decoupling technology from the target scale?


PROCESS ANALYTICS AND DOWNSTREAM PROCESSING

12:35 Chairperson’s RemarksMeisam Bakhshayeshi, PhD, Director, Process Development, Tessera Therapeutics

12:40 Downstream Processing of Viral VectorsAlois Jungbauer, PhD, Professor & Head, Biotechnology, Institute of Bioprocess Science and Engineering, University of Natural Resources and Life Sciences (BOKU)

1:10 Downstream Process Improvements in rAAV: Challenges and OpportunitiesTamara Zarubica-Zekovic, PhD, Director, Downstream Process Development, Asklepios BioPharmaceutical, Inc.Recombinant adeno-associated virus (rAAV) has emerged as a leading gene-delivery vector of choice. Despite numerous advances in the clinic, the efficiency and cost of producing rAAV drug product to meet a rapidly growing industry has significant room for improvement. AskBio has developed a model for advancing multiple clinical programs, with a focus on continuous upstream and downstream process development and improvements to large-scale manufacturing.

1:40 Optimization of an AAV Harvest Platform to Accommodate Novel, Non-Conforming AAV Capsid SerotypesCarlos A Benitez, MSEM, Scientist, Purification Process Development, Genomic Medicine Unit, SanofiThis presentation will walk through the development activities involved in assessing an AAV harvest platform fit for a new AAV capsid serotype, the associated risk/gap analysis, and the process development conducted to ensure high recovery of AAV and reduction of impurities.







Cell Cell TherapyTherapyThe Cell Therapy stream explores the critical challenges facing the analysis, quality and manufacture of cell-based therapies. Topics include product and process characterization, CMC strategies, autologous and allogeneic manufacturing strategies, automation and supply for CAR Ts and next-generation cell therapies such as NK cells, TIL and TCR-based therapies. The stream is paired with the Gene Therapy CMC and Analytics track.

Gene Therapy CMC and Analytics

Cell Therapy CMC and Analytics

Conference Programs

AUGUST 16-17

AUGUST 18-19






5th Annual


All Times EDT


MONDAY, AUGUST 16


CMC STRATEGIES FOR GENE THERAPIES9:55 Chairperson’s RemarksJames Warren, PhD, Vice President, Pharmaceutical Development, Ultragenyx Pharmaceutical

10:00 KEYNOTE PRESENTATION: Current Challenges in Gene Therapy Technical DevelopmentMark Galbraith, PhD, Former Head, Quality Control and Analytical Sciences, Spark Therapeutics

Implementation of a manufacturing process that assures a predefined quality of product is a critical requirement for the licensing and marketing of every CGT product. This presentation will discuss the current challenges in gene therapy technical development to ensure safe, well-characterized products.

10:30 FEATURED PRESENTATION: Potential Challenges Throughout the Development ProcessAndrew W. Harmon, PhD, Biologist, CMC Reviewer, Division of Cellular and Gene Therapies, Office of

Tissues and Advanced Therapies, FDA CBERThis presentation will discuss common challenges that may arise during development of Gene Therapy products. Topics will include the importance of anticipating the next steps in product development to ensure that early activities, including product characterization and assay development, can support potentially rapid clinical study progression. Regulatory mechanisms available to facilitate development of promising Gene Therapy products will also be highlighted.

11:00 Analytical Strategies for Recombinant AAV Characterization and Product ReleaseZhu Zhen Pirot, PhD, Vice President, Translational Science, Kriya TherapeuticsManufacture scale up and analytics are key challenges in AAV gene therapy development. Establishment of clear analytical strategies aligning with process & manufacture development from early translational development is critical for speed-to-clinical development. The presentation will discuss our integrated strategies and some key analytical development.

Ebene 1 ��

11:30 Vector Safety Assessment in Cell and Gene TherapyWei Wang, PhD, Director, Business Development, GeneWerkGeneWerk specializes in vector safety and integration site analysis for gene- and cell- therapy. We also offer a wide variety of custom-tailored services around safety and efficacy, such as analyses for gene-editing on/off-targets, TCR/BCR immune repertoire, or AAV impurities. Our goal is to assist clients in moving towards safer therapies.


CHARACTERIZATION AND POTENCY ASSAYS12:50 Chairperson’s RemarksMark Galbraith, PhD, Former Head, Quality Control and Analytical Sciences, Spark Therapeutics

12:55 Stability-Indicating Assays for AAV-Based VectorsMarina S. Feschenko, PhD, Director, Gene Therapy, BiogenAAV-based vectors used in Gene Therapy are new and complex modalities both in terms of structure and function. The mechanism of action of viral vectors is multifaceted, which requires matrixed approach for assessment of biological activity. This presentation will focus on stability-indicating methods for AAV-based vectors and will discuss challenges and opportunities for structure-activity relationship studies.

1:25 Potency Assay Strategy for AAV Gene TherapiesAisleen McColl-Carboni, PhD, Director, Analytical Development, Homology Medicines, Inc.AAV gene therapy products have complex mechanisms of action that pose unique challenges to potency assay development. Determining the true biological activity often requires multiple assays (i.e. a matrix approach), and the strategy for implementing these assays may evolve through the product lifecycle. This presentation will discuss phase-appropriate development and qualification of different in vitro potency assays.

1:55 TEM Analytics to Check Purity, Integrity, and Formulation Changes in the Production of Viral Vectors and VaccinesQiong Wu, PhD, Senior scientist, Electron Microscopy Services, Vironova BioAnalytics ABVironova revolutionizes transmission electron microscopy (TEM) for nanoparticle characterization in the fields of gene therapy, vaccines, and drug delivery based on image analysis. The high-resolution images can provide essential information of morphology, packaging, integrity, and purity of the sample. Our in-house image analysis software and GMP-certified laboratory provide TEM services that answer questions which arise during the production process, helping in the decision-making in viral vector development.


ANALYTICAL STRATEGIES (VIRTUAL SESSION)2:40 Characterization of DNA Starting MaterialsLawrence C. Thompson, PhD, Senior Principal Scientist, Analytical R&D, Pfizer Inc.The release testing package for plasmid DNA starting materials is more or less a platform set of assays while the amount (if any) of characterization testing is much less defined. This presentation will discuss 4 different examples of nucleic characterization tools and how they can be used to enhance the release methods and probe the quality of the starting material a little deeper than previously considered.





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3:10 Advanced Analytics for Virus-Based Gene TherapiesShibani Mitra-Kaushik, PhD, Director & Head, Bioassay & Molecular Analytical Development, Genomic Medicine Unit, SanofiVirus-based gene therapies require complex analytical methods to assess key quality attributes during process development, GMP release and stability monitoring of clinical grade product. This presentation will discuss phase appropriate paradigms for analytical method development, qualification, and implementation into gene therapy programs as they transition from research into clinical stages. Some challenges with material and time limitations and proposed resolution strategies using platform-based methods will be explored as well.









6:30 Close of Day

TUESDAY, AUGUST 17


ANALYTICAL STRATEGIES FOR GENE THERAPIES7:55 Chairperson’s RemarksAdriana Kita, PhD, Associate Director, Analytical Development, Ultragenyx

8:00 Novel Methods for AAV Product CharacterisationTony Bou Kheir, PhD, Lead Technical Scientist, Cell and Gene Therapy CatapultAs the gene therapy field continues to expand and companies increase the number of product batches manufactured per year, the burden on quality control for product release increases significantly. The presentation will showcase CGT Catapult’s advanced analytical platform for AAV product characterisation, which includes assay development for improving precision, assay automation and in-line product characterisation for closed processes.

8:30 Implementation Strategy of a Next-Generation Sequencing WorkflowJarrod Dean, Associate Director, Genomic Medicine Unit, SanofiThis presentation will include a case study detailing the validation of an NGS analysis pipeline for GMP program support, use of Agile methodology to track project progression and deliverables; and a proposed strategy to support the transfer of an NGS analysis pipeline from a development to production environment

9:00 Analytical Method for Determination of Expression of a Secondary Element in the Transgene Cassette by RT-PCRCarlos E. Peredo, PhD, R&D Associate Fellow, Cell and Gene Therapy Platform, GlaxoSmithKlineT cells transduced with self-inactivating lentiviral vectors that co-express affinity-enhanced cancer antigen T cell receptors (TCRs) and T cell function modulators may demonstrate antitumor efficacy superior to TCRs alone. This work describes an analytical method which demonstrates the expression of transgenic T cell function modulators distinctly from their endogenous counterparts for characterization of lentiviral vectors and transduced T cell products.

9:30 High Throughput Characterization of Antibody, AAV and Cell Therapy Aggregates Using the AuraBernardo Cordovez, PhD, Chief Science Officer and Founder, Halo LabsIn all biological products, distinguishing aggregated API from other particle types matters for understanding the root cause of instability. Until now, subvisible particle characterization methods have been unreliable, slow, and difficult to use across many workflows. Introducing the Aura, a 96-well, low-volume, high throughput aggregate and particle imaging system can rapidly size, count, and characterize biological particles and identify them as proteins, non-proteins, cellular aggregates, or other types of molecules.





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ANALYTICAL STRATEGIES FOR GENE THERAPIES10:40 Chairperson’s RemarksWei-Chiang Chen, PhD, Associate Director, Sanofi

10:45 Characterization of AAV Genome and Co-Packaged DNA Impurities by Next-Generation SequencingWei Zhang, Associate Director, Analytical Development, UltragenyxFor AAV gene therapy products, partial genomes and encapsidated residual DNAs impurities pose safety risk and compromise product efficacy. Their levels need to be monitored and controlled during product development. Next generation sequencing offers orthogonal approaches to traditional methods in characterizing such impurities, providing insights on genome integrity, DNA impurities levels, and size.

11:15 Improved qPCR Methods for AAV Genome Titer and Residual DNA QuantificationYu Wang, PhD, Senior Scientist, Analytical Development, BiogenqPCR method is widely used to quantify genome titer and residual DNA in AAV gene therapy products. However, the standard method requires complicated sample treatment and is known to be variable. In this presentation, we will introduce a new method with optimization of AAV sample preparation and choice of qPCR standard, which not only simplify the method, shorten the assay time, but also improve overall assay performance.

11:45 AAV Capsid Quantification Methods and Its Relevance to Other Gene Therapy Product Quality AttributesAdriana Kita, PhD, Associate Director, Analytical Development, UltragenyxCapsid titer can be measured using a variety of analytical techniques and is important for both early and late stage process development. Here, we compare methods for measuring AAV capsid titer and evaluate these measurements against other product quality attributes including genome titer and empty/full capsid ratios.

12:15 pm Process Development Strategy of Adeno-Associated Viral VectorKenneth Warrington, Senior BD Director, Strategy & Business Development, GenScript ProBioA growing number of gene therapy products are currently in preclinical or clinical trials to explore the potential in the treatment of multiple diseases. Among gene therapy vehicles, adeno-associated viral vector (AAV) is one of the most actively investigated deliver vectors with superior safety. GenScript ProBio helps solve the challenge of scalable and stable manufacturing for AAV from preclinical through IND application to clinical phases, bring excellent solutions to gene therapy.

12:30 Standardizing rAAV Production for Gene TherapyNeal Goodwin, PhD, Chief Scientific Officer, TeknovaTeknova is developing an optimized ion exchange (IEX) chromatography algorithm to purify full rAAV viral vectors that account for vector diversity. We rapidly assessed multiple parameters and created customized protocols and reagents for downstream processing. To date, we have used the formulated algorithm to develop optimal IEX protocols and ready RUO or GMP reagents for purifying multiple serotypes and payloads.



ADVANCED ANALYTICAL STRATEGIES1:55 Chairpersons’s RemarksWei-Chiang Chen, PhD, Associate Director, Sanofi

2:00 Advanced Analytics for AAV CharacterizationSantoshkumar L. Khatwani, PhD, Director, Analytical Development, Sangamo TherapeuticsThis talk will focus on advanced analytics of AAV with respect to critical quality attributes - vg titer, empty full AAV, aggregation, MS. In addition, this talk will show examples of orthogonal approaches to switch from traditional to advanced analytics for AAV.

2:30 Expedite Gene Therapy Development by Advanced Mass Spectrometry CharacterizationVictor Chen, Principal Scientist, RegenxbioGene therapy products have demonstrated a great potential to treat devastating diseases and are being extensively evaluated in clinical trials for many disease indications. The structural and biological properties of these products are complex and yet to be fully realized. Advanced mass spectrometry applications are developed to assess AAV vector quality attributes. Case studies of mass spectrometry applications in expediting gene therapeutic development will be presented.

A-GENE, QbD, MAM3:00 Update on A-Gene: Best Practices for the Manufacture of AAV VectorJosephine Lembong, PhD, Manager, Science and Industry Affairs, Alliance for Regenerative MedicineThe Cell & Gene Therapy industry is rapidly growing with more commercial product approvals anticipated each year. However, the lack of standardization and best practices around CMC programs creates hurdles to streamlined, cost-effective manufacture. Borrowing from the ‘A-Mab’ model in the monoclonal antibody field, the Alliance for Regenerative Medicine has produced a similar document titled A-Gene, a case study-based approach to integrating Quality by Design principles in gene therapy manufacturing.





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3:30 QbD for Gene TherapiesJessie Sun, PhD, Director, Ultragenyx Pharmaceutical, Inc.


4:45 Implementation and Adaptation of Multi-Attribute Mass Spectrometry for Adeno-Associated Virus (AAV) Vector CharacterizationJoshua Powers, PhD, Post-Doctoral Associate, NIST Biomolecular Measurement DivisionGene therapies possess substantial potential to be revolutionary curative treatments, but their broad implementation requires further progress in biomolecular understanding. High-resolution analytical approaches must be adopted to fully characterize these molecularly complex treatments and monitor the final drug product. A multi-attribute mass spectrometry method was developed to monitor AAV vector quality attributes and detect new peaks detection, which should be widely applicable to other vector-based modalities.


CO-PRESENTATION: IN-PERSON INTERACTIVE DISCUSSION: Gene Therapy AnalyticsWei-Chiang Chen, PhD, Associate Director, SanofiVictor Chen, Principal Scientist, Regenxbio

5:45 Close of Gene Therapy CMC and Analytics Conference





6th Annual

Cell Therapy CMC and AnalyticsOptimizing the Quality, Characterization and Manufacture of Cell-Based Therapies August 18-19

All Times EDT




CMC CHALLENGES FOR CELL-BASED THERAPIES7:55 Chairperson’s Opening RemarksJunxia Wang, PhD, Head of Analytical Development, Cell Therapy Franchise, National Resilience, Inc.

8:00 KEYNOTE PRESENTATION: CMC Shares Center Stage with Clinical Data in the Successful Development of a Cell-Based TherapyDon W. Fink, Jr., PhD, Master Practice Expert,

Regulatory, Dark Horse ConsultingFDA’s recent Complete Response Letter (CRL) for Meoblast’s remestemcel-L Biologics License Application highlights perceived increased scrutiny being given to Chemistry, Manufacturing, and Controls (CMC) during cell-based therapy development. While review of well-characterized small molecule drugs predominantly emphasizes clinical results there is growing recognition that for cell-based therapies, the more challenging issues are related to product manufacturing and quality assurance.

8:30 Management of Raw Materials for the Manufacturing of 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, quality and safety

9:00 Analytical Strategy for Key Materials and Cell Products in Gene-Edited Cell TherapiesBo Yan, PhD, Senior Scientist, Analytical Development, Beam TherapeuticsBase editing offers a fundamentally new strategy for precise gene editing. Multiple critical materials (such as gRNA, mRNA, plasmid, etc,), and delivery vehicles (such as lentivirus, adeno-associated virus AAV, and lipid nanoparticles) mean the processes and products are very complex. As a result, unprecedented analytical needs arise from such complex processes and drug products. Here, I will discuss the overall analytical development strategy in developing base editing drug candidates.

9:30 On the Importance of Monitoring Both Transfection and Transduction Efficiencies in Cell Therapy DevelopmentDerek C. Lenz, PhD, Senior Marketing Manager, BioPharma, Field Marketing, Beckman Coulter Life SciencesPertaining to cell-based therapies, monitoring transfection vector payloads has become important. While several techniques are described, studies show specific advantages conferred by Analytical Ultra-Centrifugation.In contrast, often absent is direct cell transduction monitoring; analyses do occur, but further downstream. Data are presented on the Coulter Principle as uniquely suited for transduction monitoring. Together, the two platforms lend efficiencies in cell therapy development.


STARTING MATERIALS, PROCESS CONTROL (VIRTUAL SESSION)

10:40 Standards and Measurement Control Strategies to Improve Confidence in the Characterization and Testing of Cellular Therapeutic ProductsSumona Sarkar, PhD, Biomedical Engineer, Biosystems and Biomaterials Division, Biomaterials Group, National Institute of Standards and TechnologyThe characterization and testing of cellular therapeutic products (CTPs) is a critical aspect of product development, translation and release. Here I will describe recent efforts in the standardization of the characterization and testing of CTPs. I will also describe recently published standards on cell counting as well as NIST technical programs for increasing confidence in cell count and viability assays.

11:10 Control of Critical Analytical ReagentsChristopher Bravery, PhD, Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd.The quality and performance of complex analytical reagents are arguably as critical to product quality as raw materials. Even different batches of some materials may require re-evaluation of the analytical method at some level, especially where custom made. This talk will discuss what is expected along with tips and advice on how to avoid issues.



ANALYTICAL STRATEGIES, POTENCY ASSAYS1:25 Chairperson’s RemarksDon W. Fink, Jr., PhD, Master Practice Expert, Regulatory, Dark Horse Consulting





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1:30 CMC Analytics Strategies for Cell-Based TherapiesJunxia Wang, PhD, Head of Analytical Development, Cell Therapy Franchise, National Resilience, Inc.Here, we will discuss the determination of process and method goals for the improvement of robustness of analytical method for cell-based therapies, risk assessment, specification of a design space, implementing a control strategy and continuous improvement to increase method robustness and knowledge.

2:00 Quality by Design Bioprocess Characterization for IDCT: An Allogeneic Cell Therapy for Lower Back PainDaniel Rodriguez-Granrose, Senior Bioprocess Scientist, Bioprocess Development, DiscGenics IncOur team is developing an allogeneic cell therapy (rebonuputemcel) to treat painful disc degeneration. Each production lot comes from a new donor cell line with unique quality attributes, which poses a significant challenge in the development of robust biomanufacturing practices. Here we discuss practical quality by design and process modeling techniques to build a multivariate design space with special considerations for the impact of donor variability on critical quality attributes.

2:30 Single-Cell Polyfunctional Biomarkers Enabling The Next Generation of Cellular ImmunotherapyVictor Tek, Senior Product Manager, IsoPlexis







solutions that can accommodate the rapidly evolving landscape of biopharmaceutical products while also minimizing impact on the

environment. This talk will highlight some of the major opportunities for reducing the environmental impact of bioprocessing through the application of metrics such as process mass intensity and lifecycle assessment methodology. Results will be presented from both a Genentech perspective and industry-wide perspective.


6:00 Close of Day

THURSDAY, AUGUST 19


CHARACTERIZING CELL-BASED THERAPIES 7:55 Chairperson’s RemarksDominic Clarke, PhD, ISCT Process & Product Committee Co-Chair & Chief Technology Officer, Cell and Gene Therapy, Discovery Life Sciences

8:00 Analytical Control Strategy and Characterization Approach of Novel Red Cell Therapeutic ProductsKapil Gupta, PhD, Senior Director, Cell Therapy, Analytical & Drug Product Development Rubius Therapeutics, Inc.Rubius Therapeutics is developing a novel cell therapy platform by genetically engineering donor-derived precursor cells to express therapeutic proteins inside or on the cell surface and culturing them into red blood cells, called Red Cell Therapeutics. Characterization of RCT product for CQA assessment requires a multi-pronged approach with a comprehensive analytical toolbox. Technical challenges in assessing cellular phenotype, biophysical properties, and biological activity of RCTs will be discussed.

8:30 Characterization of Genetically-Modified CD34+ Cell Drug ProductsRiccardo Biavasco, PhD, Scientist, Analytical Development, Bluebird BioCell-based gene therapy drug products require comprehensive analytical assays to characterize their safety and efficacy. This seminar will focus on the GMP release tests implemented across multiple phase I/II and III clinical trials and the additional non-GMP characterization assays performed to address heterogeneity of drug products. Correlative analyses between the results of different analytical assays will be discussed.


9:30 Vector Copy Distribution at a Single-Cell Level Enhances Analytical Characterization of Gene-Modified Cell TherapiesVincenzo Di Cerbo, PhD, Lead Scientist, Cell & Gene Therapy CatapultThe ability to deliver transgenes into the human genome using viral vectors is a major enabler of the gene-modified cell therapy field. However, controlling viral transduction is difficult and can lead to product heterogeneity, impacting efficacy and safety. We generated and qualified a novel assay to measure vector copy distribution at a single cell level and label-free transduction efficiency, and demonstrated its applicability on clinically relevant hematopoietic stem cell therapies.





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10:00 Insightful New Data on Improved Yield & Quality for AAV Manufacturing Using OsmolalityMark Rothenberg, PhD, Associate Director, Scientific Applications, Advanced InstrumentsImproving the manufacturing processes used in CGT requires an understanding of certain critical parameters. One such parameter, osmolality, has long been considered a critical tool in bioprocessing and recently has been implicated across the entire gene therapy process workflow. This seminar will focus on the impact of osmolality on improving the quality and yield of an AAV manufacturing process, showing a significant shift on the relative extracellular vs intracellular vector yield.


IN-PERSON INTERACTIVE DISCUSSION: Staging for Commercialization – Navigating the Transition from DevelopmentDominic Clarke, PhD, ISCT Process & Product Committee Co-Chair & Chief Technology Officer, Cell and Gene Therapy, Discovery Life Sciences

11:30 LUNCHEON PRESENATION: Your Future Cell Therapy Manufacturing Success-Story: The Lonza Cocoon® with Integrated Magnetic SeparationPeter Yates, PhD, Business Development & Clinical Application Manager, Lonza Personalized MedicineThe Lonza Cocoon® is a closed, automated platform for end-to-end cell therapy manufacturing. As manufacturing processes evolve, Lonza continues to upgrade the Cocoon® Platform with new functionalities. The soon-to-be-released “Integrated, in-line magnetic cell separation” will allow the automated isolation of T cells as a unit operation within the system, resulting in standardized cellular input material and greater control over closed cell therapy manufacturing processes.


MANUFACTURING CELL-BASED THERAPIES12:35 Chairperson’s RemarksRuud Hulspas, PhD, Technical Director, Process Development, Dana-Farber Cancer Institute

12:40 Towards a Robust Manufacturing Process for Autologous iPSC-Based TherapiesRuud Hulspas, PhD, Technical Director, Process Development, Dana-Farber Cancer InstituteThe Connell & O’Reilly Families CMCF at the DFCI has 15 separate ISO-7 clean rooms, 2 method development and 1 QC Lab. The facility focusses on translating prototype products into cGMP-compliant manufacturing processes. The presentation will cover a variety of IND-application and CMC requirements that are encountered during development of a manufacturing process for autologous iPSC-based therapies.

1:10 AI and Laser Processing for Scalable Generation of Autologous iPSC-Based Cell TherapiesMarinna Madrid, PhD, Co-Founder, Cellino BiotechCellino’s vision is to make personalized regenerative medicines viable at large scale. Cellino’s platform combines label-free imaging and high-speed laser editing with machine learning to automate cell reprogramming, expansion, and differentiation in a closed cassette format, enabling thousands of patient samples to be processed in parallel in a single facility.

1:40 Considerations for the Development of Gene-Edited Stem Cell TherapiesBrent Morse, Vice President, Process & Analytical Development, Vor Biopharma Inc.The era of gene editing has unlocked the potential to cure diseases with few treatment options. In particular, gene-edited stem cell therapies have shown great promise in the treatment of beta-thalassemia and sickle cell disease, and could change the treatment landscape in hematological malignancies. This talk will discuss some points to consider when developing a gene-edited stem cell manufacturing process.


MANUFACTURING CELL-BASED THERAPIES (VIRTUAL SESSION)

2:50 Challenges in Scaling up Manufacturing for Allogeneic iPSC-based TherapiesRichard J Anderson, PhD, Senior Director, MSAT, Fate Therapeutics Inc

3:20 GEN-011 PLANET Process: A Robust and Rapidly Scalable Manufacturing Process to Generate Neoantigen-Targeted Peripheral T Cells (NPTs)Manish Jain, Vice President, Pharmaceutical Sciences & Manufacturing, Genocea BiosciencesGEN-011 is an autologous, neoantigen-specific T cell therapy. GEN-011 is produced by the PLANET (Proliferation of Lymphocytes Activated by Neoantigens Endogenous in Tumors) manufacturing process, and T cells are specifically expanded on neoantigens identified by Genocea’s proprietary antigen discovery platform ATLAS™. This presentation will provide an insight into the PLANET process and go over our strategies and considerations to overcome manufacturing challenges associated with personalized therapy.






Vaccine Vaccine ManufacturingManufacturingThe Vaccine Manufacturing stream will explore the technical challenges facing the production and supply of vaccines for COVID-19 and future pandemics. Topics include manufacturing at global scale, CMC development under accelerated timelines, new technologies, ensuring product quality, and managing complex supply chains. Examples will come from novel and traditional vaccine manufacturing platforms.

Bioproduction: Scale, Bioreactors

& Digitalization

Vaccine Development and Manufacturing

Conference Programs

AUGUST 16-17

AUGUST 18-19






Inaugural

Vaccine Development and ManufacturingOptimizing the Development, Supply and Quality of Vaccines



MONDAY, AUGUST 16


mRNA VACCINE DEVELOPMENT AND MANUFACTURING (VIRTUAL SESSION)

10:00 Developing and Manufacturing mRNA Vaccines at ScaleUlrich Blaschke, PhD, Vice President, Technical Development, BioNTechmRNA is increasingly investigated as a platform

technology for multiple therapeutic applications, including as a vaccine against infectious diseases. With respect to manufacturing, mRNA has several advantages compared to other biopharmaceuticals. With the outbreak of COVID-19 in early 2020, our mRNA platform was thus ideally suited to develop an mRNA-based vaccine in “lightspeed.” In this talk, an overview about the program and the challenges that we faced will be given.

10:30 KEYNOTE PRESENTATION: Enabling Low and Middle Income Countries to Access mRNA VaccinesPhilippe-Alexandre Gilbert, PhD, Senior Program Officer, Vaccine Development and Surveillance, Bill and Melinda

Gates FoundationmRNA vaccines represent a promising alternative to conventional vaccine approaches. Unfortunately, at the current time, low-income countries (LMIC) cannot afford these high cost of goods (COGs) products, nor have the capabilities to replicate/duplicate this technology. One hopes this scenario will be different for the next pandemic, however major breakthroughs in mRNA vaccine supply model and mRNA vaccine technologies will be needed.

11:00 Formulation Strategies for COVID 19 VaccinesLee Christopher Smith, Principal Consultant, GreyRigge Associates Ltd.Live viral vaccine formulation face particular challenges including limited analytical tools and inherent variability that make evaluation difficult. While most viral vaccines are liquid presentations, product storage may include frozen, lyophilised or even dried. Properly formulated and stabilised, a product should retain potency and efficacy for years. Knowing the stressors triggering instability and designed approaches for evaluation provide a firm foundation for sound formulation and will be discussed.

11:30 Exploring New Frontiers: Empowering Manufacturing of mRNA VaccinesRatish Krishnan, Senior Strategy Consultant, Novel Modalities - Bioprocessing, MilliporeSigmamRNA technology offers a great deal of versatility and have come of age during this pandemic era. The success of mRNA-based COVID-19 vaccines has created a significant level of interest for disease prevention and treatment. Promising clinical results for mRNA vaccines against SARS-CoV-2 have implications that go far beyond the current pandemic and bode well for similar approaches in the fight against cancer, heart disease and other infectious diseases. A crucial bottleneck to overcome for mRNA vaccines would be in the manufacturing arena. This talk will explore

foundational elements of the mRNA manufacturing workflow, examine key facility design considerations and delve into possibilities of a templated process.


COVID-19 VACCINE DEVELOPMENT (VIRTUAL SESSION)

12:55 INO-4800 – A DNA Based Vaccine Against COVID-19Kate E. Broderick, PhD, Senior Vice President, R&D, Inovio Pharmaceuticals, Inc.The synthetic DNA vaccine INO-4800 is being developed as a medical countermeasure to prevent COVID-19. Clinical studies are currently ongoing and have revealed the INO-4800 induces broad immune responses which neutralize the virus. Importantly, vaccination with INO-4800 has a very favorable safety profile, and the thermostability of the synthetic DNA allows for simplified global distribution and storage.

1:25 Developing and Manufacturing mRNA VaccinesDong Shen, MD, PhD, President & CEO, RNAImmune, Inc.RNAimmune has developed a third generation mRNA vaccine which targets most known mutations. The principle of RNAimmune’s proprietary technology is the use of mRNA as a data carrier to instruct the human body to produce its own proteins capable of fighting a wide range of diseases. The company applies its technologies for the development of cancer therapies, antibody therapies, the treatment of rare diseases, and prophylactic vaccines.

1:55 pm Session Break


TOOLS TO ACCELERATE VACCINE DEVELOPMENT (VIRTUAL SESSION)

2:40 Rapid Response Vaccine Development: The Molecular Clamp Platform from the Lab Bench to Mass cGMP ProductionTrent Munro, PhD, Director, National Biologics Facility; Program Director, Rapid Response Vaccine Pipeline, Australian Institute for Bioengineering and Nanotechnology, The University of QueenslandThe COVID-19 pandemic highlighted the need for both rapid vaccine platform advancement and academic partnership with industry. Advances in genomic surveillance show in real time the virus adapting to the global population. Additional tools will be required using orthogonal approaches to the current wave of vaccines. I’ll cover the development of a molecular-clamp based SARS-CoV-2 vaccine from lab bench to commercial scale production and subsequent redesign.

3:10 Production of Premium Quality SARS-CoV2 Antigens – An Ultra-Fast Process Development ApproachGerald Striedner, PhD, Associate Professor, Biotechnology, University of Natural Resources & Life Sciences, Vienna (BOKU), AustriaThe COVID pandemic has made society aware of the importance of having appropriate concepts and strategies to prevent the spread of an infectious disease, to treat infected people and to provide preventive treatment. For the area of biotechnological production, the availability of a versatile





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toolbox comprising different expression systems and generic processing concepts, is the basis for fast and efficient development and availability of high-quality products like antigens or vaccines.









6:30 Close of Day

TUESDAY, AUGUST 17


SUPPLYING AND MANUFACTURING COVID-19 VACCINES

7:55 Chairperson’s RemarksGeorge A. Robertson, PhD, CSO, Cambra Consulting, Inc.

8:00 Pandemic Response Manufacturing Supply Chain Challenges Capacity & EconomicsAndrew Sinclair, President & Founder, BioPharm Services Ltd., United KingdomRapid development of treatments for COVID presents the supply chain challenges with the dual requirements for the supply of vaccines and therapeutic treatments. Key is the ability to rapidly manufacture these costs effectively. This talk considers how the supply chain is responding, where the capacity is coming from, how much it’s costing us, and in the end, how many people do we need treat in the coming years.

8:30 Advances in Bioprocessing and Analytics to Accelerate Vaccine ProductionAntonio Roldao, PhD, Head of Cell-Based Vaccines Development Laboratory, Animal Cell Technology Unit, Instituto de Biologia Experimental e Tecnológica (iBET)Production platforms capable of manufacturing high amounts of vaccines in short time-frames are lacking. Current limitations and complexities in vaccine manufacturing will be herein reviewed, specifically related to process development and analytics implementation, with an outlook into the future. An overview of bioprocess and analytics innovations and technologies designed at iBET to overcome production challenges will be also presented. This includes cell line development, upstream and processing processing, and analytics.

9:00 The Use of Contract Manufacturing Organizations (CMO) During the PandemicGeorge A. Robertson, PhD, CSO, Cambra Consulting, Inc.The scope of this talk will comprise a review of the challenges and opportunities for the CDMO industry as observed from an insider’s point of view as a contract manufacturing subject matter expert supporting the US government. Based upon public information, the talk will focus on the challenges, how some were overcome, and what will remain for 2022 and beyond.





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9:30 Single-use Bioreactors for rapid development of novel vaccinesAnn D’Ambruoso, Manager, Product Applications and Marketing, Applikon Biotechnology, part of GetingeSingle-use bioreactors have played a large role in rapid vaccine development and production during this pandemic. Flexible and powerful controls combined with a single-use bioreactor make experiments easier to complete under tight time constraints. Data collected from micro-scale and bench-scale bioreactor systems at Wageningen University is presented as one recent example of Applikon bioreactors’ use in vaccine development.

9:45 Streamlining the Journey from R&D to GMPRick Welch, PhD, Vice President, Development Services, CDMO, Emergent BioSolutionsFrom development to market, vaccines have been moving with considerable speed over the last two years. Continuous improvement in streamlining the development and manufacture of clinical trial material has been a key driver. This presentation reviews Emergent’s overall drug development focus and timeline reduction from project initiation to GMP manufacturing. • Integrated offering• Phase-appropriate processes & systems• Process evaluation & development scale-up• Data-driven manufacturing• Flexible technology transfer


VACCINE MANUFACTURING AND ANALYTICS10:45 Process Development and Analytical Approaches to Achieving Safety, Consistency and Quality of an Inactivated Zika Vaccine CandidateNatraj Ram, PhD, Vice President, Innovation, Bioproduction, Thermo FisherA process has been developed to produce a highly purified virus employing perfusion based cell culture and virus propagation followed by multiple orthogonal purification steps. Analytical methods have been developed for in-process monitoring and control that ensures consistent performance at each step. The process has been successfully demonstrated at-scale achieving consistent performance as well as acceptable kinetics of inactivation and completeness of inactivation.

11:15 Raman Deployment for Vaccine ProductionChristopher Mahoney, PhD, Scientist, Advanced Process Control, Johnson & Johnson Pharmaceutical R&DRaman deployment in vaccine production is viewed as an essential technology, aimed to increase overall efficiency, quality, innovation, and reliability. With Raman models in vaccine production, efficiency increases with inline process characterization of metabolites and titer in real time

which can be viewed from any network laptop/desktop. Additionally, Raman models increases quality by enabling proactive issue resolution and reducing overall risk during new product introduction and tech transfer.

11:45 A Novel Platform for Glycoengineering Bioconjugate Vaccines using E. coli as a HostChristian M. Harding, PhD, Co Founder & CEO, VaxNewMo LLCBioconjugate vaccines, consisting of polysaccharides attached to carrier proteins, are generated enzymatically using protein glycosylation systems expressed in E. coli. Using a novel bioconjugation platform, we are advancing development of bioconjugate vaccines against multiple pathogens; including, Pneumococcus, Klebsiella pneumoniae, and Group B Streptococcus.

12:15 pm Novel Tools for Potency Determinations of Multivalent VaccinesDamini Patel, Field Application Scientist, InDevR, Inc.Vaccine analysis has relied on techniques that were designed years ago and take time to execute. Influenza is a classic example with the use of SRID and subjective HA/HAI. The VaxArray platform can quantify both antigens and antibodies in a variety of samples across the vaccine development process. Simultaneous measurement of multiple antigens, from bioprocess to DS to DP, will be presented. Examples will include vaccines targeting influenza, COVID, and measles/rubella.



VACCINE PRODUCTION AND FORMULATION1:55 Chairperson’s RemarksGeorge A. Robertson, PhD, CSO, Cambra Consulting, Inc.

2:00 CO-PRESENTATION: Sterile Filtration of a Live Attenuated Viral Vaccine – New Insights Using Model Particle SuspensionsAndrew Zydney, PhD, Bayard D. Kunkle Chair & Professor, Chemical Engineering, Pennsylvania State UniversityNeil Taylor, Graduate Student of Chemical Engineering, Pennsylvania State UniversityThe production of viral vaccines is highly challenging; their size and structural complexity result in challenges not always observed in therapeutic protein manufacturing. New insights into the factors controlling sterile filtration were obtained using a model particle suspension that effectively mimics a live attenuated viral vaccine, including the role of particle concentration, membrane bubble point, and membrane morphology.





Inaugural




2:30 Downstream Processing of Enveloped VLP and Extra Cellular VesiclesAlois Jungbauer, PhD, Professor & Head, Biotechnology, Institute of Bioprocess Science and Engineering, University of Natural Resources and Life Sciences (BOKU)Separation of enveloped virus-like particles from other extracellular vesicles is a challenging separation problem due to the similarity of these bionanoparticles. Without simple and scalable methods for purification and analytics, it is difficult to gain deeper insight into their biological function. A two-step chromatographic purification method was developed.

3:00 Breaking the Cold Chain with Novel Vaccine Stabilization TechnologyMaria A. Croyle, RPh, PhD, Professor of Pharmaceutics, Department of Molecular Pharmaceutics and Drug Delivery, The University of TexasDuring the COVID-19 pandemic, vaccines were developed against a novel pathogen in record time yet were fraught with significant distribution challenges. This talk will describe a novel technology, adaptable to different vaccine candidates and its success as a needle-free method for vaccine delivery with minimal cold chain requirements. Case studies with several different vaccine platforms will be discussed.

3:30 Building the Global Vaccine Manufacturing Capacity with Flexibility and ScalabilityPeter Walters, Director of Advanced Therapies, CRBSpeed and flexibility are at the forefront of life sciences trends. Innovations like rapidly deployable adaptive facilities enable advances in treatments by allowing companies to pivot and adapt to market needs quickly. See how facility design is changing to enable the rapidly evolving life sciences industry. Learn practical tips and solutions that can support how to make these facilities financially viable and compliant.



IN-PERSON INTERACTIVE DISCUSSION: Future of Vaccine Development and ManufacturingGeorge A. Robertson, PhD, CSO, Cambra Consulting, Inc.

5:45 Close of Vaccine Development and Manufacturing Conference





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BIOPROCESS DEVELOPMENT UNDER PANDEMIC CONDITIONS

7:55 Chairperson’s RemarksJames V Blackwell, PhD, President & Principal Consultant, Windshire Grp LLC

8:00 KEYNOTE PRESENTATION: 2+1: Tackling the Pandemic Using an Accelerated, Multi-Pronged ApproachDavid Maraldo, PhD, Vice President, Global Vaccines & Biologics Commercialization, Merck & Co., Inc.

Simultaneous acquisition, development, commercialization, and manufacturing readiness of three SARS-CoV-2 biopharmaceutical candidates has fostered innovation, developed new ways of working, and stimulated new technology, all with a singular focus: speed. Use of flexible facilities, modality agnostic platform technologies, and the incorporation of process analytical technology has driven acceleration opportunities. These innovations have created a responsive framework for new product introduction, with principles that have broad, future applicability.

8:30 The Pandemic-Caused Process Changes and Lessons for the FutureJames V Blackwell, PhD, President & Principal Consultant, Windshire Grp LLCThe COVID-19 pandemic has caused significant changes, both disruptions and accelerated development. Lessons learned can inform practices from the production floor to regulatory guidance for the commercialization of future therapeutics and vaccines. This presentation looks at how organizations adapted to the unprecedented critical need to get to market with life-saving vaccines; and how process development; manufacturing/operations/facilities; partnerships; and regulatory change can accelerate development in the future.

9:00 A Single-Use Clinical Manufacturing Facility Operating Under the Realities of Impact from COVIDShawn P Allwein, PhD, Sr Dir Mfg & Operations, Biologics CMC & Specialty R&D, Teva PharmaceuticalsImpact of COVID-19 to the supply chain for raw materials and consumables created major challenges for biopharmaceutical manufacturers. Single-use facilities are by nature heavily dependent on steady supply of consumables. In this presentation, we will describe how proactive planning, strong supplier relationships, creativity to find alternatives, process flexibility and risk tolerance can work to ease the supply constraints and continue to deliver to patient needs.

9:30 CO-PRESENTATION: Process Analytics for Automated Feed Control in Upstream BioprocessingUlrike Rasche, PhD, Scientific Communications Manager, Eppendorf Bioprocess Center, EppendorfWilliam Gonzalez, Bioprocessing Field Application Specialist, EppendorfWhether it be production of antibodies, vaccines, enzymes, or other product, software is needed to monitor and control the process and analyze process data. The capabilities of the software are critical to implement inline

process analytics and to implement feedback loops for automated process control. Here we present two strategies for in line process analytics aimed at automated feed control in an upstream bioprocess: Raman analyzer integration and exhaust analyzer integration.


10:40 Bioprocess Development at Pandemic PaceStefan Wieschalka, Scientist, Early Stage Bioprocess Development, Boehringer Ingelheim Pharma GmbH & Co. KGAfter the challenging year 2020, it is evident that rapidly evolving diseases call for bioprocess development at pandemic pace. In the case study at hand, we demonstrated accelerated development of an IgG antibody production process by parallelization of early development work packages and by relying on producer pool fermentations up to Phase 1 clinical supply.

11:10 KEYNOTE PRESENTATION: CMC at the Bill & Melinda Gates Medical Research Institute: Cost to Serve Drives Manufacturing and SupplyHong Liu, PhD, Biologics Process Development and Manufacturing Leader, Bill & Melinda Gates Medical

Research InstituteGates MRI’s mission is: develop products to prevent and treat tuberculosis, malaria, enteric infections and maternal, neonatal and childhood diseases, which are major causes of mortality and inequality in low-middle income countries. Urgency, affordability and accessibility are drivers to minimize cost of manufacturing, reduce time and complexity of supply. CMC makes invaluable contributions by employing technologies to enable first-in-human quickly and also provide clinically viable and commercially attractive by proof-of-concept.

11:40 Cell-Free Plasmid DNA Manufacturing ServiceShigemasa Sasaki, CBO/COO, OriCiro Genomics IncOriCiro® Cell-Free cloning system wipes the bottleneck of conventional E. coli cloning and It just two steps in vitro.OriCiro® ‘s enterprise solution for Cell-Free Plasmid DNA Manufacturing Service has capable to shows in the following benefit. • Rapid : Reaction by several hours, isothermal and enzymatic reaction,• Safety : No endotoxin, No GMO, No antibiotics and antibiotic-resistant

genes and• Process enhancement : Significant downsizing for upstream and

downstream facility



SCALE-UP AND MANUFACTURING OF AAVs1:25 Chairperson’s RemarksJeffrey Hung, PhD, Chief Commercial Officer, Vigene Biosciences, Inc.

1:30 Design, Manufacturing and Analytics of New AAV Reference Standards – A Case StudyJeffrey Hung, PhD, Chief Commercial Officer, Vigene Biosciences, Inc.AAV Reference materials for gene therapy has been lacking, resulting in poor inter-lab and inter-product comparability. Vigene has developed a standardized production protocol and analytics panel for AAV1, AA2, AAV5,





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AAV6, AAV8 and AAV9 empty and full capsids to facilitate the testing of AAV gene therapy products. The production process and analytical results will be discussed. Some lessons learned from this year-long endeavor will also be shared.

2:00 Optimisation and Scale-Up of Suspension-Based Manufacturing Processes for AAV ProductionHelen Young, PhD, Senior Scientist II, Advanced Therapeutics, CPIDeveloping high-yielding, robust, scalable and commercially viable processes to manufacture sufficient quantities of viral vectors presents challenges. This work focused on development of two suspension-based AAV production processes, scaled from shake flasks to ambr250, 10L, 50L and 200L stirred tank bioreactors. Scale-up presented challenges yet through high-throughput optimisation utilising a DoE approach, performed using the ambr250, high-yielding and scalable processes were developed with titres maintained up to 200L scale.

2:30 Scaling AAV and Cell Production to Manufacturing Scales with a Novel Intensified Adherent Cell Culture SystemJohn Yoshi Shyu, PhD, Director, Scientific Applications and Technical Support, Americas, Corning Incorporated - Life SciencesPressure to reduce time-to-market and production cost of viral vectors and cell-based therapeutics is driving demand for technologies that combine the greater yield efficiency and viable cell harvest of adherent platforms with the scale and automation of suspension systems. Dr. Shyu will present a new intensified FBR technology that has demonstrated a multi-fold increase in viral vector yield per cm2 and greater than 90% transfection rate and cell harvest efficiency.









6:00 Close of Day

THURSDAY, AUGUST 19


INNOVATIONS IN BIOPRODUCTION I - BIOREACTORS, GREEN BIOTECH, MACHINE LEARNING AND DIGITAL

TWIN (VIRTUAL SESSION)8:00 Implementation of Custom Single-Use 2L Bioreactors in Upstream Process DevelopmentBalrina Gupta, PhD, Associate Principal Scientist, Biological & Sterile Product Development, Merck Research LabsEvolution of 2-L single-use bioreactor design customization and assessment, with performance comparison to traditional glass bioreactors, will be presented. Multiple cell lines, different media and modes of operation were examined to support implementation of single use across pipeline projects.

8:30 Scale-Down of a Microbial Fermentation Process to Bottles and 250mL Single Use Bioreactors to Enable Collection of Process Characterization DataMatthew Woodling, Associate Principal Scientist, Vaccine Bioprocess R&D, Merck & Co Inc.Bioprocess development requires multiple process scales to enable the collection of large data sets that represent the commercial scale process of the product. This presentation reviews the scale down of a microbial fermentation process to the Sartorius Ambr 250mL single use bioreactor system and the associated time and cost savings when generating process characterization data at this smaller scale.

9:00 am Coffee Break in the Exhibit Hall with Poster Viewing

9:30 Application of Machine Learning Methods to Pathogen Safety Evaluation in Biological Manufacturing ProcessesShyam Panjwani, PhD, Senior Data Scientist, Bayer Healthcare PharmaceuticalsThe production of human monoclonal antibodies is not free of viral contamination risk due to adventitious agents in raw materials, cell substrates and environment. Such risk is generally mitigated by a series of time and resource expensive unit operations. Machine learning methods





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can potentially help streamline the development and optimization of viral clearance steps. The evaluation of machine learning methods for viral clearance operations is the focus of this study.

10:00 Digital Twin for Biotech ProcessesSandrine Dessoy, Senior Manager, Tech R&D, GlaxoSmithKlineA digital twin is a real-time digital replica of a physical device that communicates with it. A digital twin predicts the evolution of its corresponding physical asset and to reach this goal it utilizes state-of-the-art mechanistic and/or hybrid models, advanced sensors, connected objects and artificial intelligence. This talk is concerned with the development of digital twins in GSK for development and control of vaccines production process.

10:30 Green Biotechnology with Microalgae: Always Almost ThereJulian Rosenberg, PhD, Associate Director, Center for Biopharmaceutical Education & Training (CBET), Albany College of Pharmacy & Health SciencesMicroalgae are versatile photosynthetic organisms poised to become a sustainable platform for therapeutics, vaccines, biofuels, and plant-based protein. However, bioprocessing challenges continue to limit production at commercial scales. While the field has benefitted from advances in bioreactor design, strain selection, and bioengineering, the viability of algae-based products seems to always be just out of reach. This presentation reviews R&D accomplishments in algal biotechnology and identifies major hurdles to successful commercialization.






forward?



INNOVATIONS IN BIOPRODUCTION II - MODELING, AUTOMATION & SINGLE-USE SYSTEMS (IN-PERSON

SESSION)12:35 Chairperson’s RemarksEugene Tung, PhD, Executive Director, Manufacturing IT, Merck & Co., Inc.

12:40 Mechanistic Modeling and Parameter-Adaptive Nonlinear Model Predictive Control of a MicrobioreactorMoo Sun Hong, PhD, Postdoctoral Associate, Chemical Engineering, Massachusetts Institute of TechnologyA mechanistic model is presented for the spatiotemporal transport of oxygen through a gas-permeable membrane to the cells within a microbioreactor. The model is used to design an on-line estimator for the oxygen uptake rate of the cells, the specific cell growth rate, and the specific oxygen uptake rate. The estimates are fed to a model predictive control formulation showing improved spatial control of dissolved oxygen during cell growth.

1:10 Development of a Practical Approach for Evaluation of Single Use Fermenters with Application in Diagnostic IndustryYongxue Ding, PhD, Principal Scientist, Biologics Process Design R&D, Abbott Diagnostics Division, Abbott LaboratoriesWe will present a method through generating the ratio (%) of k_L a alike between SIP and SUF. Two scenarios were analyzed, one at low demand standard density growth, and the other at high density growth. The results matched with performance of SUF to SIP very well. This enables a practical approach for evaluation of SUF as replacement of SIP for production of biologics in diagnostic business.

1:40 Plug and Play Automation for BioprocessingEugene Tung, PhD, Executive Director, Manufacturing IT, Merck & Co., Inc.The BioPhorum Plug and Play Standard was conceived and developed through an industry-wide collaboration to enable the rapid deployment of modular equipment in biopharmaceutical facilities. The standard, co-developed by equipment providers, automation suppliers, and end-users, provides a common structure for defining an equipment’s automation capability. It utilizes existing and proven industry communication standards, eliminating the need for custom communication interfaces. Eliminating custom interfaces can save weeks in deploying automated equipment.





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INNOVATIONS IN BIOPRODUCTION III - DIGITALIZATION, REAL-TIME MONITORING & PAT

(VIRTUAL SESSION)2:50 Real-time Monitoring of AAV Production Culture System by Digital Differential Holographic MicroscopyDainan Mao, PhD, Scientist II, Ultragenyx PharmaceuticalBesides conventional offline analytical testing for AAV production at bench scale, a digital differential holographic microscopy system was developed to monitor suspension cell culture systems. Key process parameters including viable cell density and transfection/infection efficiency were determined at real-time for the production process. This process analytical technology (PAT) holds promise for future upstream process optimization for gene therapy products.

3:20 Automating Upstream: Implementing Advanced Process Control, Predictive Modelling and Online PAT Tools in Mammalian Cell Culture ProcessesBethany Kerr, Team Leader - Upstream Development, CPIAn improved understanding of the culture environment within bioreactors is essential for developing robust, high yielding processes. We will explore the usage of advanced process control and predictive modelling in mammalian cell cultures to automate and optimise bioreactor feeding and control strategies. A series of case studies will cover topics including utilising integrated PAT tools for automated at-line feeding and using machine learning to control and refine bioreactor set points.

3:50 Digitalization, Data Enablement and Analytics for Accelerated Bioprocess DevelopmentJun Huang, PhD, Senior Director, Data Enablement and Analytics, Regeneron PharmaceuticalsDigitalization, data enablement and analytics are key drivers to accelerate bioprocess development. In this talk, the operationalization of our digital/data strategy through a structured and agile program delivery will be discussed, covering 1) unified data management and IT/OT infrastructure to improve data access, sharing and integration, and 2) analytics and control capabilities to increase process visibility, comparability and predictability, as well as productivity and quality.






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