Therapeutic and Biomedical Applications of CRISPRs, ZFNs, TALENs, AAVs and other Genome Engineering Technologies March 18-19, 2015 • Hilton Boston Back Bay • Boston, MA Accelerate Your Therapeutics to Market and Improve Drug Discovery Efficiencies Learn about therapeutic applications of genome editing in cardiovascular disease, gene therapy, stem cell therapy, CART cell immunotherapy, hemophilia B, Parkinson’s disease and other advances in in vivo genome editing Receive an in-depth update on the latest technology advances and applications of CRISPR-Cas9 including a discussion of off-target issues, delivery to cells/tissues, improving specificity and multi-genic alterations Apply genome editing as a research and drug discovery tool in target identification and validation, genome-scale screening, gene targeting, cell line validation and transgenic animal models Compare the advantages and disadvantages of CRISPRs, ZFNs, and TALENs for various applications and discover ways to navigate the IP landscape of genome editing technologies Register Early for Best Rates www.IBCLifeSciences.com/GenomeEditing Genome Editing Applications Genome Editing Applications Silver Sponsors: Media Partners: Organized by: Development and Applications of CRISPR-Cas9 for Genome Editing Keynote Presentations Radical Genome Editing George Church, Ph.D., Robert Winthrop Professor of Genetics, Harvard Medical School and MIT Health Sciences and Technology Feng Zhang, Ph.D. Investigator, McGovern Institute for Brain Research and Core Faculty Member, The Broad Institute of MIT and Harvard
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Therapeutic and Biomedical Applications of CRISPRs, ZFNs, TALENs, AAVs and other Genome Engineering TechnologiesMarch 18-19, 2015 • Hilton Boston Back Bay • Boston, MA
Accelerate Your Therapeutics to Market and Improve Drug Discovery Efficiencies Learn about therapeutic applications of genome editing
in cardiovascular disease, gene therapy, stem cell therapy, CART cell immunotherapy, hemophilia B, Parkinson’s disease and other advances in in vivo genome editing
Receive an in-depth update on the latest technology advances and applicationsof CRISPR-Cas9 including a discussion of off-target issues, delivery to cells/tissues, improving specificity and multi-genic alterations
Apply genome editing as a research and drug discovery tool in target identification and validation, genome-scale screening, gene targeting, cell line validation and transgenic animal models
Compare the advantages and disadvantagesof CRISPRs, ZFNs, and TALENs for various applications and discover ways to navigate the IP landscape of genome editing technologies
Register Early for Best Rates www.IBCLifeSciences.com/GenomeEditing
Development and Applications of CRISPR-Cas9 for Genome Editing
Keynote Presentations
Radical Genome Editing
George Church, Ph.D., Robert Winthrop Professor of Genetics, Harvard Medical School and MIT Health Sciences and Technology
Feng Zhang, Ph.D. Investigator, McGovern Institute for Brain Research and Core Faculty Member, The Broad Institute of MIT and Harvard
Register Early for Best Savings • www.IBCLifeSciences.com/GenomeEditing • 800-390-4078
IBC’s Genome Editing Applications conference will showcase the most up-to-date therapeutic and biomedical applications emerging using CRISPRs, ZFNs, TALENs, AAVs and other genome engineering technologies. From validation of targeted cell lines and development of transgenic animal models to therapeutic uses in cell therapy, gene therapy and immunotherapy, this event will also highlight applications in drug target identification/validation and lead discovery and explore how these technologies are being used for whole-genome and functional screening as well as basic research applications.
A special emphasis on CRISPR will highlight the design of novel CRISPR constructs and discuss practical aspects of CRISPR including off-target effects, how to improve specificity and delivery to specific cell types, including potential applications in multi-genic alterations. By attending this applications-focused conference you will learn about advances in all of the latest genome editing technologies, including comparisons, advantages and disadvantages of each. You will also learn how to position your company for success in genome editing in the face of the still-evolving IP landscape of CRISPR and other genome editing approaches.
It’s a fact – attendees walk away with the most value when they experience it with fellow peers – so bring a colleague and save! We have a special offer for groups of 3 or more to save up to $500 off the standard rate. The earlier your group registers, the more you save. Contact our group sales representative at 646-895-7445 to get your team registered today.
Present A Poster ...Enhance Your Conference Experience
Sharing your research with your peers could lead to exciting opportunities for you to advance your career… and it can help justify the time and cost of your attendance at the conference. The deadline to submit an abstract is February 18, 2015. For complete details and pricing, please visit ww.IBCLifeSciences.com/GenomeEditing.
Bring a Colleague and Save!
“A conference not-to-be-missed for anyone actively involved in genome editing
or interested in using genome editing technologies”Inès Royaux, Ph.D., Senior Scientist, Janssen R&D, a division of Janssen Pharmaceutica NV, Belgium
Therapeutic, Drug Discovery & Research ApplicationsTherapeutic, Drug Discovery & Research Applications● Keynotes
Two world-renowned experts in genome editing
● FDA & NIH Perspective Perspectives on genome editing from the division of cellular and gene therapies
● Hot-Topic Panels• The IP landscape of CRISPR
and genome editing technologies
• Comparing the advantages and challenges of different genome editing technologies
● 18 Case Studies of therapeutic and drug discovery applications
● Industry Application Presentations from Bluebird Bio, Editas, GlaxoSmithKline, Cellectis, Sangamo, Shire, Caribou, Horizon Discovery, Pfizer, Medimmune, AstraZeneca, Janssen and Merck
March 18-19, Boston
For up-to-date program information and new abstracts, visit: www.IBCLifeSciences.com/GenomeEditing
7:00 Registration and Coffee 8:00 Chairman’s Welcome and Opening Remarks
Rob Howes Ph.D., Associate Director, HTS, Antibody Discovery and Protein Engineering, Medimmune, United Kingdom
Therapeutic Applications of Genome Editing
8:15 Engineering the Genome and Epigenome for Gene Therapy and Programming Cell PhenotypeGenome engineering tools, including zinc finger proteins, TALEs, and the CRISPR/Cas9 system, can be used to both edit gene sequences and epigenetic marks and control the expression of endogenous genes for applications in medicine and basic science. For example, we have used each of these tools for gene editing to restore the expression of the dystrophin protein that is mutated in cells from Duchenne muscular dystrophy patients. In other studies, we have developed tools for gene regulation and epigenome editing that can be applied to cell reprogramming or elucidating the role of genomic regulatory elements. These technologies have the potential to transform strategies for disease modeling, drug screening, and regenerative medicine. Charles Gersbach, Ph.D., Assistant Professor, Biomedical Engineering and Co-Director, Center for Biomolecular and Tissue Engineering, Duke University
8:45 Experimental and Therapeutic Applications of CRISPR-Cas9 in Cardiovascular Disease Genome editing offers the ability to rapidly interrogate the functions of novel genes and loci uncovered in human genetic studies such as genome-wide association studies. We are employing CRISPR-Cas9 to study novel cardiovascular genes both in traditional animal models such as mice and in human pluripotent stem cell-derived cellular models, as well as to explore therapeutic approaches (e.g., targeting the PCSK9 cholesterol gene).Kiran Musunuru, M.D., Ph.D., Assistant Professor, Department of Stem Cell and Regenerative Biology, Harvard University
9:15 The MegaTAL Nuclease Platform for T cell and Hematopoietic Stem Cell TherapiesSuccessful development of nuclease-based cellular therapeutics requires effective, safe and flexible gene editing approaches. We developed the megaTAL nuclease platform to combine the flexibility of TAL-based DNA recognition with the specificity of homing endonucleases. Bluebird bio is using custom megaTAL nucleases to create novel stem cell and T cell therapies.Alexander (Sasha) Astrakhan, Ph.D., Senior Scientist I, Research, Bluebird Bio
9:45 Networking Refreshment Break and Poster & Exhibit Viewing 10:15 Advancing the CRISPR/Cas9 Technology Platform for
Therapeutic ApplicationsGenome editing technologies, including the CRISPR/Cas9 system, enable molecular modifications to treat the underlying cause of genetic diseases. Key to the successful clinical translation of CRISPR/Cas9 systems is the optimization of the technology within the context of specific therapeutic applications. This presentation will focus on Editas’ approach to improving both activity and specificity of CRISPR/Cas9-mediated gene editing in parallel with the development of delivery solutions for therapeutic applications.David Bumcrot, Ph.D., Senior Director, Molecular & Cell Biology, Editas Medicine
10:45 Applications of Genome Editing Technologies in Drug Discovery and Stem Cell Gene TherapiesThe presentation will be focused on perspectives of genome editing technologies in drug discovery and gene therapy. A few examples will be outlined to illustrate specific applications of the technologies in establishing cellular models for target validation studies and for compound screens, together with our clinical stage programs in stem cell gene therapy. Recent results from a comparison of the ZFN and CRISPR technologies will also be discussed.Quinn Lu, Ph.D., Head, Exploratory Cell Biology, GlaxoSmithKline
11:15 Multiplex Genome Editing as a Platform for “Off-the-Shelf” Adoptive CAR T-cell ImmunotherapiesCellectis has developed a universal platform for generating chimeric antigen receptor (CAR)-redirected allogenic T-cells from third-party healthy donors using TALEN™. Nuclease mediated inactivation of the TCRalpha and CD52 genes abrogates the potential for graft-versus-host disease and renders T-cells resistant to the lymphodepleting agent alemtuzumab. Such allogeneic “off-the-shelf” CAR T-cell product will permit a wider application of CAR technology and potentially lead to a new paradigm in cancer treatment.Julianne Smith, Ph.D., Group Leader, CAR Development, Cellectis Therapeutics, France
11:45 TAL-Based Editing of iPSCs for the Study of Neurodegenerative Diseases Due to the inherent biological variability between cell lines of different backgrounds, the study of subtle phenotypic changes associated with disease states when using iPSC derived materials requires isogenic control lines for proper comparison - that is, cell lines that differ in a defined location but that are otherwise identical at the genetic level. This presentation will describe the approaches we’ve taken to modifying genes involved in Parkinson’s Disease and related disorders. For example, modifying the glucocerebrosidase gene in iPSCs using TAL-based engineering technology presented several challenges, including the presence of an adjacent and highly homologous pseudogene. Initial phenotypic comparisons between the patient derived, TAL-edited lines that we have generated will be presented.Kurt Vogel, Ph.D., Associate Director, External R&D, ThermoFisher Scientific
12:15 Luncheon followed by Dessert and Poster & Exhibit Viewing
Featured Presentation 1:30 Genome Editing with Zinc Finger Nucleases
The ability to engineer precise modifications of the human genome would revolutionize our therapeutic approach to monogenic diseases. This possibility is now being realized via zinc finger nucleases (ZFNs). ZFNs are customizable, sequence-specific endonucleases that can edit the genome at any user-chosen location. This talk will describe recent clinical results with ZFN-modified CD4 T-cells as well as preclinical proof-of-concept studies towards development of CCR5-disrupted stem cells as potential HIV treatments.Philip D. Gregory, Chief Scientific Officer, Sangamo BioSciences, Inc.
2:00 Genome Editing with Group II Introns, ZFNs, CRISPR, and Alternative Donor DNA FormatsOver the last decade, group II introns, zinc finger nucleases (ZFNs) and CRISPR/Cas systems have enabled highly efficient genome editing in a variety of bacterial and mammalian organisms. The pace of new applications has accelerated significantly over the last year due the fortuitous combination of simplicity, portability, and efficacy associated with CRISPR systems. A brief overview of these three genome editing methods will be given along with data on the latest developments in lenti-CRISPR screening of pooled gRNA libraries and alternative donor DNA formats.Gregory D. Davis, Ph.D., R&D Manager, Molecular Biotechnology, Sigma-Aldrich Corporation
2:30 ZFN-Mediated Gene Editing for the Potential Treatment of Hemophilia BZinc Finger Nucleases (ZFNs) can be engineered to site-specifically modify the human genome. We present a novel use of this ZFN gene editing platform to insert a correct copy of a human Factor IX transgene in to the liver in vivo. Using this targeted gene addition strategy, a single administration should be sufficient, to enable therapeutic and potentially lifelong production of the clotting factor for the treatment of Hemophilia B, a rare blood clotting disease.Matthew Chiocco, Ph.D., Senior Scientist, Discovery Biology & Translational Research, Shire
3:00 Networking Refreshment Break and Poster & Exhibit Viewing
Keynote Presentation 3:30 Development and Applications of
CRISPR-Cas9 for Genome Editing The RNA-guided nuclease Cas9 from the microbial CRISPR adaptive immune system has emerged as an exciting tool for precise genome editing in eukaryotic cells. This presentation will discuss the technical development as well as current and future applications and challenges.Feng Zhang, Ph.D., Investigator, McGovern Institute for Brain Research and Core Faculty Member, The Broad Institute of MIT and Harvard
WEDNESDAY, MARCH 18, 2015
Register Early for Best Savings • www.IBCLifeSciences.com/GenomeEditing • 800-390-4078
8:00 Chairman’s RemarksMarcello Maresca, Ph.D., Associate Principal Scientist, Discovery Sciences, AstraZeneca, Sweden
Latest Advances and Practical Aspects of Genome Editing Technologies
8:15 NEW DATA Crisper Designs for Cas9-mediated Genome EngineeringRNA-guided Cas9 endonucleases from CRISPR-Cas prokaryotic immune systems are widely used for programmable genome engineering. Cas9 is harnessed for multiple different applications such as research, target screening, strain engineering, and therapeutics. This talk will address improvements to the Cas9 platform necessary to realize the full potential of these varied applications, including a discussion of off-target issues, delivery to cells/tissues, improving specificity, multi-genic alterations and more.Rachel Haurwitz, Ph.D., President and CEO, Caribou Biosciences, Inc.
8:45 The Innovative Genomics Initiative: Working to Advance Genome Editing Research and Technology in Both the Academic and Commercial Sectors The Innovative Genomics Initiative is a new endeavor whose mission is to advance genome editing research and technology in the academic and commercial sectors. The IGI’s flagship projects promote technology development, basic biological research, and clinical applications of next-generation genome editing and regulation. We are additionally building outreach programs and exploring new models to encourage and accelerate entrepreneurship via training at the interface of basic and applied research.Jacob Corn, Ph.D., Scientific Director, Innovative Genomics Initiative
9:15 CRISPR Evolved: Combining the Power of CRISPR with the Specificity of rAAV for Improved Targeted Gene EditingHorizon will present data showcasing the systematic improvement of their cell line engineering platform using combination technologies (CRISPR & rAAV), as well as targeted cell line enrichment, negative selection strategies, vector optimization and automated experimental design using powerful bioinformatics tools (with our partners Desktop Genetics).
Chris Thorne, Ph.D., Gene Editing Community Specialist, Horizon Discovery, United KingdomVictor Dillard, Chief Operating Officer, Desktop Genetics, United Kingdom
9:45 Using ZFN and CRISPR to Generate Animal Models for Drug DiscoveryRecent advances in genome editing have greatly accelerated and expanded the ability to generate animal models. These tools allow generating mouse models in condensed timeline compared to that of conventional gene-targeting knock-out/knock-in strategies. Moreover, the genome editing methods have expanded the ability to generate animal models beyond mice. In this talk, we will discuss the application of ZFN and CRISPR to generate various animal models for drug discovery programs.Myung Shin, Ph.D., Principal Scientist, Biology-Discovery, Genetics and Pharmacogenomics, Merck Research Laboratories
10:15 Networking Refreshment Break and Poster & Exhibit Viewing
Keynote Presentation10:45 Radical Genome Editing
Our ability to view and alter biology progresses exponentially, outpacing electronics -- synthesizing billions of base pairs of DNA on chips and genome editing millions of new organisms per day -- moving from correlational to causality studies (using the shareable human genomics resource http://Personalgenomes.org). Genome editing also enables “radical” (i.e. genome-scale or ecosystem-scale) applications: humanization-xenotransplantation, de-extinction, gene-drives, resistance to all viruses, recoding new amino acids...George Church, Ph.D., Robert Winthrop Professor of Genetics, Harvard Medical School and MIT Health Sciences and Technology
11:30 Nucleic Acid Delivery Systems for RNA therapy and Gene EditingHigh throughput, combinatorial approaches have revolutionized small molecule drug discovery. Here we describe our work on high throughput methods for developing and characterizing RNA delivery and gene editing systems. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated and screened for their ability to delivery RNA, both in vitro and in vivo. A number of delivery formulations have been developed with in vivo efficacy, and show potential therapeutic application for the treatment of genetic disease, viral infection, and cancer.Daniel G. Anderson, Ph.D., Associate Professor, Department of Chemical Engineering, David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology
12:00 Panel Discussion The Intellectual Property and Business Strategy
Landscape of Genome Editing Technologies• Current status of the IP landscape of CRISPR and other technologies • Overview of the current business and partnering landscape in genome editing• Freedom of use considerations for genome editing technologiesPanelists:Ellen Law, Senior Counsel, Director, Intellectual Property, The Broad InstitutePatricia Granahan, Shareholder, Wolf GreenfieldSamuel Hasson, Ph.D., Principal Investigator, Pfizer Neuroscience Chris Thorne, Ph.D., Gene Editing Community Specialist, Horizon Discovery
12:30 Luncheon followed by Dessert and Poster & Exhibit Viewing
Genome Editing Applications in Drug Discovery and Screening
1:45 RNAi and the Genome Editing Revolution: Technology Synergy to Improve the Power of Functional Genomic StudiesFunctional genomic screens are a powerful method to map biological pathways and validate novel targets. These screens conducted with arrayed or pooled-reagents have been dominated by RNAi technologies. Now, as CRISPR/Cas9-based platforms emerge, their synergy with the versatile RNAi offers an unprecedented workflow to improve the quality of functional genomic campaigns.Samuel Hasson, Ph.D., Principal Investigator, Pfizer Neuroscience
THURSDAY, MARCH 19, 2015
Moderator: Charles Gersbach, Ph.D., Assistant Professor, Biomedical Engineering and Co-Director, Center for Biomolecular and Tissue Engineering, Duke UniversityPanelists:Jacqueline Corrigan-Curay, M.D., Director, Office of Biotechnology Activities, Office of Science Policy, Office of the Director, National Institutes of Health Gregory D. Davis, Ph.D., R&D Manager, Molecular Biotechnology, Sigma-Aldrich Corporation Philip Gregory, Ph.D., Senior Vice President and Chief Scientific Officer, Sangamo Biosciences, Inc.Daniel Takefman, Ph.D., Chief, Gene Therapy Branch, Division of Cellular & Gene Therapies, OCTGT, CBER, FDAJulianne Smith, Ph.D., Group Leader, CAR Development, Cellectis Therapeutics, FranceFeng Zhang, Ph.D., Investigator, McGovern Institute for Brain Research and Core Faculty Member, The Broad Institute of MIT and Harvard
Panel Discussion 4:15 Comparing the Advantages and Challenges
of Different Genome Editing Technologies: Technical and Regulatory Perspectives• What is the best system for generating your knock-outs?• How to improve specificity of guide RNAs?• How to check for off-target effects?• Are there systems in other species that might be better
than the standard CRISPR system?• Latest advances in guide RNA design • What technologies are on the horizon?
WEDNESDAY, MARCH 18, 2015 (continued)
Venue and AccommodationsHilton Boston Back Bay40 Dalton St., Boston, Massachusetts, 02115, USA TEL: 617-236-1100 or toll free at 800-HILTONS (445-8667) FAX: 1-617-867-6104Please call the hotel directly at the number above before February 20, 2015, or until sold out. Please mention IBC’s Genome Editing Applications to receive the reduced room rate. The hotel will require a first and last night’s non-refundable deposit at the time the reservation is made.
*Academic/Government rates are extended to full-time employees of government, universities and university-affiliated hospitals only.
Special Room Ratefor Attendees:
$209/night + tax
For up-to-date program information and new abstracts, visit: www.IBCLifeSciences.com/GenomeEditing
Present a Poster All poster presenters must be registered conference attendees. Poster fees are additional: Vendors/Supplier: $125 Pharma/Biotech: $125 Academic/Government: FREE. Vendor rate is for exhibiting/sponsoring companies and/or posters that upon review are of commercial/product focus.
Registration PoliciesPlease visit www.IBCLifeSciences.com/GenomeEditing for the complete registration, payments, cancellation, substitutions and special needs policies.Program content and speakers subject to change.
2:15 Genome-scale CRISPR-Cas9 Screening in Human CellsThe simplicity of programming the CRISPR (clustered regularly interspaced short palindromic repeats)-associated nuclease Cas9 to modify specific genomic loci suggests a new way to interrogate gene function on a genome-wide scale. We recently created a genome-scale CRISPR-Cas9 knockout (GeCKO) library with 64,751 unique guide sequences targeting 18,080 genes and have delivered this library in a lentiviral format for negative and positive selection screening in human cells. Neville Sanjana, Ph.D., Simons Postdoctoral Fellow, Laboratory of Feng Zhang, The Broad Institute of Harvard and MIT
2:45 Application of CRISPR and Other Gene Targeting Technologies in Biologics Drug DiscoveryThere are several challenges specific to the development of therapeutic biologics such as antibodies, recombinant proteins and peptides. At MedImmune we are using a range of gene targeting technologies to develop a range of isogenic cell lines to be used at key points in the drug discovery process. We will present data on a range of endogenous reporter cell lines and how these can be used to identify novel biologics.Rob Howes Ph.D., Associate Director, HTS, Antibody Discovery and Protein Engineering, MedImmune, United Kingdom
3:15 Networking Refreshment Break and Last Chance for Poster & Exhibit Viewing
3:45 ObLiGaRe: Highly Efficient Targeted Integration by Non Homologous End Joining (NHEJ) Using ZFNs, TaleNs and CRISPR/CASThe precise integration of foreign DNA is an essential step for the generation of cellular and animal disease model and for gene therapy. Genome editing tools (ZFNs, TaleNs, CRISPR/CAS) greatly facilitate this process but current protocols based on homologous recombination have several limitations. We developed a new method (ObLiGaRe) that overcomes these limitations by using the efficient and constitutive pathway of NHEJ.Marcello Maresca, Ph.D., Associate Principal Scientist, Discovery Sciences, AstraZeneca, Sweden
4:15 Accelerating Drug Discovery Using Genome Editing TechnologiesIn order to speed up the drug discovery process, Janssen R&D has adopted gene editing technologies in its early phase activities. Here we will present two case studies: use of gene-edited cell lines to study off-target effects of kinase inhibitors and generation of modified iPS cell lines for disease modeling.Inès Royaux, Ph.D., Senior Scientist, Janssen R&D, a division of Janssen Pharmaceutica NV, Belgium
4:45 Applications of CRISPR-Cas9 Gene Editing in Drug Target ValidationIdentification and validation of drug-resistant mutations can provide important insights into the mechanism of action of a compound. Here we demonstrated the feasibility of such an approach in mammalian cells using next-generation sequencing of drug-resistant clones and CRISPR-Cas9–mediated gene editing. Our study suggests that CRISPR–Cas9 system could become an important part of an integrated approach to target validation in mammalian cells.Mi Cai, Ph.D., Senior Scientist, Pfizer Neuroscience
5:15 Close of Conference
Sponsorship and Exhibit Opportunities – Reserve Your Exhibit Booth TodayIf you are a CRO or technology provider who offers genome editing and/or CRISPR, TALEN or ZFN-related technologies, reagents and services, you will not want to miss this conference. Attendees of this conference are looking for genome editing and genome engineering solutions in the following areas: bioinformatics and software tools, automated and high-throughput systems, animal models/transgenic animals, next-generation sequencing tools, synthetic biology tools, gene synthesis, stem cells, genomic services, cell line development and more.
For more information, please contact Kristen Schott • [email protected] • 508-614-1239Silver Sponsors: Bronze Sponsors:
