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February 23-25 2016 | Boston, USA • Improve Target Specificity & Efficiency of Genome Editing • Enhance Drug Discovery & Screening Applications • Design & Develop Clinical Therapies 2 nd Annual Rachel Haurwitz Caribou Biosciences Jon Moore Horizon Discovery Alexandra Glucksmann Editas Medicine Emmanuelle Charpentier Max Planck Institution, Umeå University George Church Harvard Medical School Rodger Novak CRISPR Therapeutics This is designed to describe the profound impact CRISPR is having on basic research and therapeutic development. It will explore the research tools, bioinformatics and expertise needed to make the most of this breakthrough technology. Eric Rhodes, Horizon Lead Partner Additional Event Partners Tel: +1 212 537 5898 | Email: [email protected] RESEARCHED & DEVELOPED BY: crispr-congress.com

2nd CRISPR Congress Boston, 23-25 February 2016

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Page 1: 2nd CRISPR Congress Boston, 23-25 February 2016

February 23-25 2016 | Boston, USA

• ImproveTargetSpecificity&EfficiencyofGenomeEditing• EnhanceDrugDiscovery&ScreeningApplications• Design&DevelopClinicalTherapies

2ndAnnual

RachelHaurwitzCaribou Biosciences

JonMooreHorizon Discovery

AlexandraGlucksmannEditas Medicine

EmmanuelleCharpentierMax Planck Institution, Umeå University

GeorgeChurchHarvard Medical School

RodgerNovakCRISPR Therapeutics

This is designed to describe the profound impact CRISPR is having on basic research and therapeutic development. It will explore the research tools, bioinformatics and expertise needed to make the most of this breakthrough technology.

EricRhodes,Horizon

LeadPartner AdditionalEventPartners

Tel: +1 212 537 5898 | Email: [email protected]

RESEARCHED & DEVELOPED BY:crispr-congress.com

Page 2: 2nd CRISPR Congress Boston, 23-25 February 2016

Welcome to Those Dedicated to CRISPR

2. Engineer the next generation of humanized animal models & cell lines for improved disease modeling and preclinical predictability

2.Harness CRISPR technology for more accurate gene editing in non-eukaryotic bacterial and plant cells

5 Key Benefits to Takeaway1.Discover innovative ways to measure and

optimize specificity of the CRISPR/Cas9 system in order to reduce off target effects

1.Effectively adopt the lentiviral delivery system to optimize genome wide targeting from basic research to drug discovery

3.Develop strategies to perform large scale genome wide screening from basic research to disease target identification

3.Utilize CRISPR/Cas9 to develop robust cell-based cancer immunotherapies including CAR-Ts and TCRs

4.Learn how to independently design and optimize CRISPR with novel emerging enzymes (Cpf1) to enhance the precise nature of genome editing

4.Understand the regulatory requirements for CRISPR/Cas9 mediated therapeutic development as it is poised to demonstrate clinical utility

5. Enhance the delivery and efficiency of CRISPR/Cas9 in primary cells

5. Harness CRISPRi to elevate the regulation of gene expression through epigenetic modifications

5 Key Ideas to Implement

Tel: +1 212 537 5898 Email: [email protected]

crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing

2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

Achieve the Full Potential of CRISPR Genome Editing to Ensure Accuracy & SuccessOptimizeyourCRISPRDesigntoEfficientlyPowerNovelApplicationsinDrugDiscovery,ScreeningandTherapeuticDevelopment

The 2nd Annual CRISPR Congress will enhance the basic research, drug discovery and therapeutic applications of CRISPR technology by overcoming key specificity, efficiency and delivery challenges needed to improve the precise editing and repair of the genome.

With the superior applications of CRISPR showing no signs of relenting, join the leading CRISPR figureheads as they reveal advanced methodology, strategies and clinical timelines that maximize the power of precision genome editing and fulfill its revolutionary potential.

As well as applying CRISPR to optimize the custom development of stable cell lines & disease models for

target identification and enhanced drug discovery, CRISPR Congress 2016 pioneers the translation of CRISPR-based gene therapies into a clinically relevant transformative therapy.

As CRISPR continues to create a sea change for genetic research, not to mention the next class of enzymes on the horizon, join the CRISPR Congress to deepen your understanding, minimize off-target effects and elevate delivery mechanisms for therapeutic use. There is a a key opportunity for you to network and build future collaborations with fellow peers to optimize the application of CRISPR gene editing technology and transform your research to the next level.

Why the 2nd CRISPR Congress Should Be in Your Calendar

Page 3: 2nd CRISPR Congress Boston, 23-25 February 2016

Speaker Faculty

2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

Tel: +1 212 537 5898 Email: [email protected]

crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing

RachelHaurwitzPresident & CEOCaribouBiosciences

AlexandraGlucksmannChief Operating OfficerEditasMedicine

EmmanuelleCharpentierProfessorMaxPlanckInstitutionforInfectionBiology, MIMSUmeåUniversity

GeorgeChurchProfessor of GeneticsHarvardMedicalSchool

JonMooreCSOHorizonDiscovery

JohnFederAssociate Director of Genome BiologyBristol-MyersSquibb

ChadCowanAssociate Professor, Harvard Stem Cell Institute, Broad Institute HarvardUniversity

TJCradickHead of Genome EditingCRISPRTherapeutics

JoelKlappenbachDirector of Applied GenomicsMerck

GregoryDavisR&D Manager, Molecular BiotechnologySigma-Aldrich

CharlesGersbachAssociate Professor, Director, Center of Biomolecular & Tissue Engineering DukeUniversity

DavidRootSenior Director Genetic Perturbation Platform and the Functional Genomics Consortium BroadInstitute

ChannabasavaiahGurumurthyAssistant Professor-Genetics, Director- Mouse Genome Engineering Core Facility, UniversityofNebraskaMedicalCenter

GregGocalSenior Vice President R&DCibus

BenjaminKleinstiverPostdoctoral Fellow, Keith Joung’s Group, MassachusettsGeneralHospital&HarvardMedicalSchool

EmilyLeproustCEOTwistBioscience

LorenzMayrVP & Global Head, Reagents & Assay DevelopmentAstraZeneca

JohnDoenchAssociate Director, Genetic Perturbation Platform, MITHarvardUniversity

JenniferBermanStaff Scientist, Applications, Digital Biology CenterBio-RadLaboratories

RodgerNovakCEOCRISPRTherapeutics

JonathanGootenbergFeng Zhang’s Lab, Broad Institute, MITHarvardUniversity

JasonPotterSenior Scientist & ManagerThermoFisherScientific

ShawnZhouSenior ScientistGenscript

WenXueAssistant Professor, RNA Therapeutics Institute, UniversityofMassachusetts

AnjaSmithDirector of R&DDharmacon,GEHealthcare

MarkBehlke,Chief Scientific Officer IntegratedDNATechnologies,Inc

DanielAndersonSam Goldblith Associate ProfessorMassachusettsInstituteofTechnology(MIT)

TomBarnesCSO IntelliaTherapeutics

Speakers all gave excellent presentations - perfect balance of viewpoints on leveraging CRISPR technology. I enjoyed the format and structured networking sessions were well thought out – I appreciated the opportunity to connect with experts in this emerging field.

Previous CRISPR Congress Attendee WarpDriveBioLLC

Page 4: 2nd CRISPR Congress Boston, 23-25 February 2016

Conference Day One

8.00 Registration,Breakfast&Networking9.00 Chair’sOpeningRemarks

CRISPRtheSuperior:ARevolutionThatSpeedsOn

9.10 KeynoteInterviews:OptimizingCRISPRDesigntoEfficientlyPowerNovelApplicationsinDrugDiscovery,ScreeningandTherapeuticDevelopment

The panel will discuss and answer the following questions: • How has the novel CRISPR enzyme Cpf1 enhanced the application

CRISPR technology?• With continued innovation - Will CRISPR always be at the top or are there

better alternatives emerging to drive gene engineering?• How has the industry tried to enhance CRISPR/Cas9 mediated gene-editing in

primary cells?• How is the industry addressing the ethical challenges surrounding

genetic editing of germline cells in order to be diligent whilst pushing scientific boundaries?

• From a therapeutic perspective, how close are we to demonstrating clinical utility and what have been the lessons learning in preparing CRISPR-based gene therapies for human trials?

EmmanuelleCharpentier,Professor, MaxPlanckInstitutionforInfectionBiology,MIMS,UmeåUniversity

AlexandraGlucksmann, Chief Operating Officer,EditasMedicine

RodgerNovak,CEO, CRISPRTherapeutics

RachelHaurwitz,President & CEO, CaribouBiosciences

TomBarnes,CSO, IntelliaTherapeutics

10.00 CRISPR/Cas9–ASwissArmyKnifefortheIdentificationandValidationofNovelDrugTargets• Learn to accurately analyze data from screens and to functionally

validate them• Genome wide screening for gain of function or loss of function screening,

either in exploring the developmental state for basic biology or for screening for therapeutic targets for diseases (drug development)

• Discover how to establish CRISPR libraries for target identification

JonMoore, CSO, HorizonDiscovery

10.30 MorningRefreshments&SpeedNetworking

MeasuringandOptimizingSpecificityoftheCRISPR/Cas9System

11.30 KeynotePresentation:TheCRISPR-Cas9RevolutioninGenomeEngineering:LessonsLearnedfromBacteria• Addressing the origins of CRISPR-Cas9 • Discover the mechanisms of CRISPR-Cas9 and to highlight the evolution

of CRISPR-Cas9

EmmanuelleCharpentier,Professor, MaxPlanckInstitutionforInfectionBiology,MIMS,UmeåUniversity

12.15 RewritingDNASynthesis• Highlighting the innovative silicon-based platform that is revolutionizing

DNA synthesis• Addressing how the platform delivers the power of scale, reduces price

and turn-around time through the miniaturization of reaction volumes by a factor of 1000

• Allowing design on a grander scale by simplifying experiments through circumventing the need to clone; whether it is genes, ready-to-use oligonucleotide pools or variant libraries

EmilyLeproust,CEO,TwistBioscience

12.25 BeyondCas9:DiscoveringNewToolsforPrecisionGenomeEditing• Using smaller, easier to deliver Cas9 orthologs enables new translational

applications for genome editing• Cas9 is just one of many programmable nucleases in nature with gene

editing potential• The RNA-guided endonuclease Cpf1 expands the genome engineering toolbox

and allows for allele-specific targeting

JonathanGootenberg,Feng Zhang’s Lab, HarvardUniversity

Tel: +1 212 537 5898 Email: [email protected]

crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing

2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

Tuesday February 23rd 2016

Page 5: 2nd CRISPR Congress Boston, 23-25 February 2016

16.55 BreakoutRoundtables:AdvancingCRISPRTechnologytoTheNextLevel

Discover multiple perspectives on the key issues and applications in the revolutionary CRISPR field by joining roundtable discussions, specifically designed so you can learn from your fellow gene-editing peers.

Moderators:JoelKlappenbach,Director of Applied Genomics, MerckDanielAnderson,Sam Goldblith Associate Professor,MassachusettsInstituteofTechnology(MIT)

Discovering how to perform high throughout

genome screenings in the development of

CRISPR libraries

5.Collaborating to

develop and define protocols for gene

editing in non-eukaryotic cells

4.Advancing non-viral delivery

methods to progress CRISPR-therapeutics – how can you refine methodology to overcome this key hurdle

of CRISPR therapeutic development in the context

of clinical utility

3.Harnessing CRISPRi technology to fast-track

development of therapies – ensuring sequence

specific control of gene expression

2.Evaluating and combining

different methods to enhance specificity

of CRISPR to minimize unwanted

off-target effects

1.

12.55 Ultra-SensitiveQuantificationofGenomeEditingEventsbyDropletDigitalPCR• Genome editing events can be rare and difficult to quantify, with frequencies

<5% in some targeted cell types• Genome editing methods development and optimization would benefit from a

sensitive, rapid-readout tool for edit validation and off-target detection• Droplet Digital PCR (ddPCR) enables sensitive (<0.1%), precise absolute

quantification of NHEJ and HDR alleles in a rapid, high-throughput format

JenniferBerman, Staff Scientist, Applications, Digital Biology Center, Bio-RadLaboratories

13.25 NetworkingLunch

14.25 EnsuringSuccessfulTranslationfromthePreclinicaltoClinicalStagesUsingCRISPR/Cas9• Addressing the safety aspects involving specificity

• Learning from other gene editing tools in the clinic including TALENs and ZFNs

AlexandraGlucksmann,Chief Operating Officer, EditasMedicine

14.55 GenomeEditingSolutions-NewWorkflowToolsforGenomeEditing• High Efficiency editing in difficult cell lines using cas9 mRNA or RNPS

• Improved delivery using Neon electroporation or Lipofectamine CRISPRMAX

JasonPotter,Senior Scientist & Manager, ThermoFisherScientific

15.25 HowEpigenomeEditingCanBeUsedtoReprogramCellPhenotypeforDiseaseModelingandRegenerativeMedicine• Insight into how epigenome editing tools have been developed to perturb gene

regulatory elements

• Annotating the function of regulatory elements can be used to discover new drug targets, diagnostics, and strategies for personalized medicine

CharlesGersbach,Associate Professor, Director, Center of Biomolecular & Tissue Engineering, DukeUniversity

15.55 AfternoonRefreshments&PosterSession

16.25 BeyondtheBreak:OptionsforDonor-DNADrivenGenomeEditingwithHighFidelityandEfficiency• Donor DNA formats and their impact on DNA repair rates

• Learn to manipulate DNA repair outcomes among diverse cell types

• Strategies for pooled screens using CRISPR and donor-DNA libraries

GregoryDavis,R&D Manager, Molecular Biotechnology, Sigma-Aldrich

17.55 Chair’sClosingRemarks JonMoore,CSO, HorizonDiscovery

18.00 EveningDrinksReceptionHostedbyHorizonDiscovery

Tel: +1 212 537 5898 Email: [email protected]

crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing

2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

Page 6: 2nd CRISPR Congress Boston, 23-25 February 2016

Tel: +1 212 537 5898 Email: [email protected]

crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing

2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

Conference Day Two

8.00 Breakfast&Networking

9.00 Chair’sOpeningRemarks

9.10 GenomeEngineeringTechnologiesandApplications• Using Cas9 to mutate up to 62 sites in one cell simultaneously

• Applications include xenotransplantation, and gene drives to eliminate malaria

GeorgeChurch,Professor of Genetics,HarvardMedicalSchool

AdvancingLargeScaleGenomeWideScreening

9.40 WorkingTogethertoUnlockthePotentialofPreciseGenomeEditing• Case studies where CRISPR/Cas has been the key enabler for doing

drug discovery

• Understanding the evolving role of CRISPR/Cas9 technology for target discovery, target validation and mechanistic studies

• Leveraging external academic and industrial research collaborations for genome-wide target discovery

LorenzMayr,VP & Global Head, Reagents & Assay Development, AstraZeneca

10.10 InnovativeTraitDevelopmentToolsinPlantBreedingwillbeCrucialforDoublingGlobalAgriculturalProductivityby2050• Rapid Trait Development System (RTDS™) employs Gene Repair

OligoNucleobases (GRONs) to make defined spelling changes in genomic DNA

• We report that RTDS can significantly improve the outcome of double strand break activity by reliably inducing precise and targeted nucleotide spelling changes closely aligned to the cut site

• Our work demonstrates the significance of gene editing to rapidly, precisely and reliably improve cro performance to develop any trait in commercially relevant crop varieties

GregGocal, Senior Vice President, Research & Development, Cibus

10.40 OpportunitiesandChallengesofAppliedGenomeEngineeringTechnologiesinthePharmaceuticalResearchSetting• Genome engineering is advancing at an incredible pace however,

applications across the types of translational and predicative cell models now preferred for drug target discovery remains a challenge

• What areas of the drug discovery process will genome engineering have the biggest and most immediate impact

• How does the pharmaceutical industry evaluate and implement a technology that is evolving so rapidly

JohnFeder,Associate Director of Genome Biology,Bristol-MyersSquibb

11.10 MorningRefreshments

11.40 ImprovingCRISPR-Cas9GeneKnockoutwithaValidatedGuideRNAAlgorithm• Development of an algorithm for improved CRISPR RNA functionality

and specificity

• Utility of a synthetic two-RNA approach for high-throughput arrayed gene knockout

• Importance of rigorous alignment tools for CRISPR RNA specificity

AnjaSmith,Director of R&D,Dharmacon,GEHealthcare

Wednesday February 24th 2016

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HarnessingCRISPRTechnologyinDiseaseandPreclinicalModeling

12.10 OptimizingBioinformaticToolstoImproveGuideDesignandtoEnhanceSpecificity• Optimize CRISPR/Cas systems to drive gene editing• Utilize CRISPR corrects the underlying disease-causing mutations• Harness bioinformatics and design strategies to improve and

ensure specificity• Discovering the use of new CRISPR systems

TJCradick, Head of Genome Editing, CRISPRTherapeutics

12.40 GenomeEditing:FromModelingDiseasetoNovelTherapeutics• The use of genome editing to generate isogenic cell lines with and

without disease mutations for rigorous human disease modeling, either by introducing mutation(s) into wild-type human pluripotent stem cell (hPSC) lines or by “curing” the mutation(s) in patient-specific human induced pluripotent stem cells (hiPSCs)

• Utilizing genome editing tools to rapidly and efficiently generate knockout or mutant alleles of several genes in human pluripotent stem cells to differentiate both targeted lines and isogenic control lines into various cell types relevant to metabolic disorders

• Learn how to assess which system is more efficient in human pluripotent stem cells by targeting the same genomic sites in more than a dozen genes using both TALENs and CRISPRs in the same hESC lines or hiPSC lines

• Sequencing genome-edited clones to compare the rates of off-target effects produced by the TALEN and CRISPR systems

ChadCowan,Associate Professor, Harvard Stem Cell Institute, Broad Institute,HarvardUniversity

13.10 ApplyingGenCRISPREfficiently–PioneeringtoMakeGenome-EditingEasy• GenCRISPR services cover mammalian and bacterial cell

line services• GenCRISPR-related reagents and making research easy through

one-stop service solution

ShawnZhou,Senior Scientist,GenScript

13.20 NetworkingLunch

14.20 TippingtheCRISPRNHEJ/HDRImbalance:StrategiesforEfficientGenerationofLarge-Size-DNAKnock-inAnimalModels• Latest developments in enhancing insertion of larger DNA

molecules using HDR-dependent and HDR-independent mechanisms

• The CRISPR strategies that help rapid development of humanized animal models

ChannabasavaiahGurumurthy,Assistant Professor-Genetics, Director- Mouse Genome Engineering Core Facility, UniversityofNebraskaMedicalCenter

14.50 PrecisionCancerMouseModelsThroughGenomeEditingwithCRISPR-Cas9• Address how CRISPR can speed up the generation of precision

cancer models• How these models can be used to understand the evolution and

progression of individual tumors• Identify new strategies for cancer treatment

WenXue,Assistant Professor, RNA Therapeutics Institute, UniversityofMassachusettsMedicalSchool

Tel: +1 212 537 5898 Email: [email protected]

crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing

2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

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MaximizingPrecisionforNovelApplications

15.20 IncreaseEfficiencyofGenomeEditingUsingtheAlt-R™CRISPR-Cas9System• Improving on-target CRISPR-Cas9 genome editing performance

while also reducing cell toxicity and eliminating innate cellular immune response as compared to in vitro transcribed guide RNA alternatives

• A discussion on challenges and potential pitfalls of genome editing with guidance towards successful CRISPR-Cas9 editing experiments

MarkBehlke,Chief Scientific Officer,IntegratedDNATechnologies,Inc

15.50 AfternoonRefreshments

16.20 ApplicationofCRISPRTechnologiestoGeneticScreens• As a powerful tool for large scale screens how do CRISPR libraries

perform in actual screens?

• What factors are experimentally shown to be important?

• What are strengths and limitations of CRISPR libraries and screens?

DavidRoot,Senior Director,Broad Institute Genetic Perturbation Platform and the Functional Genomics Consortium, BroadInstitute

16.50 GeneticScreenswithCRISPR-Cas9:OptimizingOn-TargetActivityandAvoidingOff-TargetEffects• Considerations for library design to optimize on-target activity

• Detection and avoidance of potential off-target activity

• Comparison of CRISPR to RNAi

JohnDoench, Associate Director, Genetic Perturbation Platform, MIT, HarvardUniversity

17.20 ExpandingandImprovingtheGenome-WideSpecificitiesofCRISPR-Cas9Nucleases• The targeting range of Cas9 can be expanded by engineering

variants that can target previously inaccessible sites

• The genome-wide specificity of Cas9 can be improved with novel evolved variants

• GUIDE-seq can be used to define the genome-wide specificities of Cas9 variants

BenjaminKleinstiver,Postdoctoral Fellow, Keith Joung’s Group,MassachusettsGeneralHospital&HarvardMedicalSchool

17.50 Chair’sClosingRemarks

17.55 EveningDrinksReceptionHostedbySigma-Aldrich

Tel: +1 212 537 5898 Email: [email protected]

crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing

2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

Page 9: 2nd CRISPR Congress Boston, 23-25 February 2016

Workshop A

Tel: +1 212 537 5898 Email: [email protected]

crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing

2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

CRISPR technology has rapidly changed the face of biological research. In particular, precision genome editing has been so readily adopted it has now become routine for many labs. One key application of CRISPR is in the discovery and functional understanding of genomics through high-throughput screening. In this interactive workshop, attendees will capitalize lessons learned in order to:

• Develop protocols to carry out large scale genome screens to better understand gene interactions in disease

• Learn from a range of applications to optimize the development of CRISPR libraries

• Optimize data analysis of genome screens for target identification in drug discovery

CRISPR/Cas9-mediated genome editing has become the preferred tool of choice when it comes to efficiently and easily manipulating the genome with strong specificity. As such, a rapidly adopted application has emerged to create more physiologically relevant and predictable in vivo disease models to better understand the complexity of human disease. In this interactive workshop, attendees will discover solutions and answers to help them:

• Outline the latest in developments in the CRISPR-mediated generation of disease models

• Overcome the current limitations of using CRISPR for disease modeling

• Harness CRISPR gene editing technology to develop humanized animal models, particularly for emerging immunotherapies

• Address and discuss case studies to standardize protocols to utilize CRISPR for in vivo models

PerformingHighThroughoutGenomeScreeningsforDevelopingCRISPRLibrariesDate:ThursdayFebruary25th2016|Time:9.00am–12.00pm

DevelopingMorePredictableDiseaseModelsWithCRISPR:FromBasicResearchtoTargetIdentificationandDrugDiscoveryDate:ThursdayFebruary25th2016|Time:1.00pm–4.00pm

TJ Cradick, PhD has studied the different families of nucleases that enable genome editing and gene therapy, such as for correcting mutations that cause diseases. Recent work has focused on CRISPR/Cas nucleases (see publication list). Previously we developed and studied TAL Effector Nucleases (TALENs), and Zinc Finger Nucleases (ZFNs). Many of these studies in academia or industry included measuring and improving specificity. TJ co-authored manuscripts detailing our bioinformatics web tools: ZFN-Site, PROGNOS, SAPTA and COSMID (CRISPR off-target).

WorkshopLeaderTJCradick,Head of Genome Editing, CRISPRTherapeutics

CB Gurumurthy obtained a faculty position at the University of Nebraska Medical Center (UNMC), Omaha in 2007. He is currently an assistant professor in Developmental Neuroscience Department and serves as the Director of UNMC Mouse Genome Engineering Core Facility. He is actively pursuing research in developing newer geneome editing technologies in addition to developing widely-usable animal models for basic and drug discovery research.

WorkshopLeaderChannabasavaiahGurumurthy, Assistant Professor-Genetics, Director- Mouse Genome Engineering Core Facility, UniversityofNebraskaMedicalCenter

Workshop B

Page 10: 2nd CRISPR Congress Boston, 23-25 February 2016

CRISPR Partners

Sigma-AldrichSigma-Aldrich is proud to offer its newest line of genome editing tools, Sigma CRISPRs, to the global research community. Sigma CRISPRs offer rapid, reliable and reproducible results – everything you need for gene editing experiments. Sigma-Aldrich also offers the CRISPR Core Partnership Program providing scientists and core facilities with world class service and a diverse portfolio of innovative CRISPR reagents, with the support of an industry-leading bioinformatics engine. The Cell Design Studio team at Sigma Aldrich, utilizes CRISPR, ZFN, and shRNA technologies to rapidly and efficiently generate model cell lines to be used in basic research, cell-based assays, target validation and much more.

www.sigmaaldrich.com

ThermoFisherScientificThermo Fisher Scientific Inc. (NYSE: TMO) is the world leader in serving science, our mission is to enable our customers to make the world healthier, cleaner and safer. We help our customers accelerate life Sciences research, solve complex analytical challenges, improve patient diagnostics and increase laboratory productivity. Through our four premier brands Thermo Scientific, Life Technologies, Fisher Scientific and Unity Lab Services we offer an unmatched combination of innovative technologies, purchasing convenience and comprehensive support.

www.thermoscientific.com

Program Partner

Program Partner

Bio-RadBio-Rad Laboratories, Inc. designs, manufactures, and distributes a broad range of innovative tools and services to the life science research and clinical diagnostics markets. Founded in 1952, Bio-Rad has a global team of more than 7,750 employees and serves more than 100,000 research and industry customers worldwide through the company’s global network of operations. Throughout its existence, Bio-Rad has built strong customer relationships that advance scientific research and development efforts and support the introduction of new technology used in the growing fields of genomics, proteomics, drug discovery, food safety, and medical diagnostics.

www.bio-rad.com

Program Partner

Tel: +1 212 537 5898 Email: [email protected]

crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing

2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

HorizonDiscoveryHorizon Discovery combines long scientific heritage in translational research with GENESIS™, a precision gene editing platform incorporating rAAV, CRISPR and ZFN technologies. Horizon supplies genetically-defined cell lines, gene-editing tools and services, custom cell line generation, molecular reference standards, and contract research services to approaching 1,000 academic, clinical and biopharmaceutical organisations.

www.horizondiscovery.com

Lead Partner

Page 11: 2nd CRISPR Congress Boston, 23-25 February 2016

CRISPR Partners

Dharmacon–partofGEHealthcareDharmacon has revolutionized the field of RNA synthesis with the introduction of 2’-ACE synthesis chemistry since 1995. As leaders in custom RNA synthesis, Dharmacon was an early participant in the newly discovered field of RNA interference, and contributed several key scientific findings. Dharmacon RNAi products were some of the first commercially available. This leadership has continued through technical advances in bioinformatics, and chemical modifications to improve performance. Today, our areas of research and research tools have expanded to support all aspects of RNAi interference; siRNA, lentiviral shRNA, tools for microRNA research, and whole-genome scale libraries for RNAi functional screens of genes, microRNAs and long non-coding RNAs. Dharmacon also has the largest collection of cDNAs and ORFs commercially available.

In addition, Dharmacon offers a unique set of CRISPR-Cas9 gene editing tools. The Dharmacon CRISPR-Cas9 platform greatly simplifies the workflow of permanently knocking out genes. Our approach includes pre-designed, ready-to-use DNA and RNA components and enables fast assessment of multiple target sites per gene for multiple genes. We offer CRISPR Guide RNA, high quality, ready-to-use lentiviral and synthetic reagents to guide Cas9 cleavage; Cas9 Nuclease for your cell type to ensure robust Cas9 expression or explore DNA-free options; CRISPR Controls and Detection Primers essential to assessment of CRISPR-Cas9 genomic editing experiments; CRISPR-Cas9 Pooled sgRNA or arrayed crRNA for high-throughput gene editing studies.

www.dharmacon.gelifesciences.com

Program Partner

CibusCibus has developed plant and microbial platforms enabling it to become a world leader in precision gene editing, generally, and advanced non-transgenic breeding, specifically. The Rapid Trait Development System (RTDS™), its proprietary technology for non-transgenic breeding, enables site-specific edits of native genes with no introduction of foreign DNA. The precise and predictable outcomes can replicate products developed using traditional mutagenesis and are identical to those that could occur in nature, given enough time. Consequently, these plants are not considered transgenic, the key attributes of GMO crops.

www.cibus.com

Program Partner

IntegratedDNATechnologiesIntegrated DNA Technologies (IDT) is a leader in the manufacture and development of products for the research and diagnostic life science market. The largest supplier of custom nucleic acids in the world, IDT serves academic research, biotechnology, and pharmaceutical development.

IDT products support a wide variety of applications, including next generation sequencing (NGS), genome editing, DNA amplification, SNP detection, microarray analysis, expression profiling, gene quantification, and synthetic biology. Platform-independent NGS products and services are available in addition to DNA and RNA oligonucleotides, qPCR assays, siRNA duplexes, reagents for CRISPR-Cas9 genome editing, and custom gene synthesis services.

IDT has an active research and development division that supports product development, and participates in corporate and academic collaborations. IDT also supports the education of the next generation of scientists through our regular sponsorship of the international Genetically Engineered Machine (iGEM) competition, and other scientific outreach activities.

www.idtdna.com

Program Partner

TwistBioscienceOur expertise is synthetic DNA. We have developed a manufacturing process featuring a 10,000-well silicon platform capable of producing synthetic biology tools. Our platform overcomes the current inefficiencies of synthetic DNA production, and enables cost-effective, rapid and high-quality synthetic gene production. The Twist Bioscience platform has the potential to accelerate the development of personalized medicine, sustainable chemical production, improved agriculture production as well as new applications such as in vivo diagnostics, biodetection and data storage.

www.twistbioscience.com

Spotlight Partner

Tel: +1 212 537 5898 Email: [email protected]

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2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

Page 12: 2nd CRISPR Congress Boston, 23-25 February 2016

ChardanCapitalMarketsChardan Capital Markets, LLC is a privately held investment banking firm with a focus on micro, small, and mid-cap markets within the healthcare and technology sectors. Our full range of services includes capital raising, merger and acquisition advisory, strategic advisory, equity research, institutional trading, and market making. Having established a successful record of completing complex and challenging deals, we’ve become a leading authority on capital solutions for emerging growth companies.

www.chardancm.com

Hosting Lunch Partner

ERSGenomicsERS Genomics was formed to commercialize the foundational CRISPR-Cas9 intellectual property held by Dr. Emmanuelle Charpentier. Licenses are available for research and sale of products and services across multiple fields including: research tools, kits, reagents; discovery of novel targets for therapeutic intervention; cell lines for discovery and screening of novel drug candidates; GMP production of healthcare products; production of industrial materials such as enzymes, biofuels and chemicals; and synthetic biology.

www.ersgenomics.com

Networking Partner

CRISPR Partners

AdvancedAnalyticalTechnologiesAdvanced Analytical Technologies, Inc. (AATI), founded in 1998 and located in Ankeny, Iowa, is a world leader in multi-channel (parallel) capillary electrophoresis. The company’s products are designed to improve processes within the life science, agricultural, molecular diagnostics, biofuels, biotechnology, and pharmaceutical industries. Its award winning instrument, the Fragment Analyzer™ is the premier instrument to automate the analysis of nucleic acid fragments and smears. Designed to improve laboratory workflow and decrease time to results, the Fragment Analyzer can dependably and reliably assess gene editing events generated through CRISPR/Cas9 mutagenesis using a streamlined heteroduplexing and cleavage process for ultra-sensitive detection of fragments and digital data outputs with specially designed software to aid in analysis. Additionally, Reagent Kits are available to accurately qualify and quantify nucleic acid raw materials like genomic DNA and RNA and analyze NGS library preparation. The Fragment Analyzer is the premier instrument to automate analysis of nucleic acids.

www.aati-us.com

Exhibitor

MTI-GlobalStemMTI-GlobalStem develops high-quality, standardized stem cell reagents and innovative technologies for life science and neurobiology research. Our products include primary and iPSC-derived neural cells, neuronal culture media and supplements, pluripotent stem cells and stem cell culture media, feeder cells, and transfection reagents for hard-to-transfect cells. Our latest transfection products are optimized for large plasmid delivery of CRISPR/Cas9 vectors into a range of cell types, including hard-to-transfect primary cells.

www.mti-globalstem.com

Exhibitor

GenScriptGenScript is the leading gene, peptide, protein and antibody research partner for fundamental life science research, translational biomedical research, and early stage pharmaceutical development. Since our establishment in 2002, GenScript has exponentially grown to become a global leading Contract Research Organization that provides services and products to scientists in 86 countries worldwide

www.genscript.com

Spotlight Partner

Tel: +1 212 537 5898 Email: [email protected]

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2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

Page 13: 2nd CRISPR Congress Boston, 23-25 February 2016

Code:5792* All discount offers (including team discounts) require payment at the time of registration to receive any discount. ‘Early Bird’ discounts require payment at time of registration and on or before the cut-off date to receive any discount. All discount offers cannot be combined with any other offer. The conference fee includes lunch, refreshments and course documentation. The fee does not include travel or hotel accommodation.

Full payment is due on registration. Cancellation and Substitution Policy: Cancellations must be received in writing. If the cancellation is received more than 14 days before the conference attendees will receive a full credit to a future conference. Cancellations received 14 days or less (including the fourteenth day) prior to the conference will be liable for the full fee. A substitution from the same organisation can be made at any time.

Changes to Conference & Agenda: Hanson Wade reserves the right to postpone or cancel an event, to change the location or alter the advertised speakers. Hanson Wade is not responsible for any loss or damage or costs incurred as a result of substitution, alteration, postponement or cancellation of an event for any reason and including causes beyond its control including without limitation, acts of God, natural disasters, sabotage, accident, trade or industrial disputes, terrorism or hostilities.

Data Protection: The personal information shown and/or provided by you will be held in a database. It may be used to keep you up to date with developments in your industry. Sometimes your details may be obtained or made available to third parties for marketing purposes. If you do not wish your details to be used for this purpose, please write to: Database Manager, Hanson Wade, 4th Floor, 52 Grosvenor Gardens, London, SW1W 0AU

HansonWadeLimited.RegisteredinEngland&Wales.CompanyNo:6752216

Terms & Conditions

Register

Event Prices

Pricing & VenueHyattRegencyBostonOneAvenuedeLafayetteBoston,Massachusetts,USA,02111

Accommodation: Overnight accommodation is not included in the registration fee, however accommodation options will be sent out with your confirmation email upon registering.

Academic&Not-for-ProfitPricing

PackageRegister&

PaybeforeFridayJanuary22nd2016

StandardPrices

GoldPackage: Conference + 2 Workshops $2297 (save $300)

$2397 (save $200)

SilverPackage:Conference + 1 Workshop $1898 (save $200)

$1998 (save $100)

BronzePackage:Conference Only

$1499 (save $100) $1599

WorkshopEach: $499

IndustryPricing

PackageRegister&

PaybeforeFridayJanuary22nd2016

StandardPrices

GoldPackage: Conference + 2 Workshops $3697 (save $400)

$3797 (save $300)

SilverPackage:Conference + 1 Workshop $3098 (save $300)

$3198 (save $200)

BronzePackage:Conference Only

$2499 (save $200) $2699

WorkshopEach: $699

Tel: +1 212 537 5898 Email: [email protected]

crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing

2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016

•10%discount–3 delegates •15%discount–4 delegates •20%discount–5 or more delegates

Please note that discounts are only valid when three or more delegates from one company book and pay at the same time.

Team Discounts*

crispr-congress.com/registerTel:+1 212 537 5898 Email:[email protected]

Mail:Hanson Wade 4th Floor, 52 Grosvenor Gardens, London, SW1W 0AU