Gene Editing Workshop...April 28, 2019 Washington, D.C. 5 Washington Hilton Gene Editing Workshop...

Gene Editing Workshop

Washington, D.C.

April 28, 2019

Washington Hilton

Lincoln

April 28, 2019 Washington, D.C. 1 Washington Hilton


Table of ContentsWorkshop Sponsors . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2

Program Schedule . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5

Co-Chairs . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7

Committee Listing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7

Speaker Listing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7

Speaker Bios . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8

Disclosure of Relevant Financial Relationships . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14

Notes . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15



Gene Editing Workshop SponsorsThe American Society of Gene & Cell Therapy is honored to acknowledge the following organizations for their sponsorship of the Gene Editing Workshop:

Workshop Sponsors



Program

10 AM – 10:20 AM

Introduction to Gene EditingMorgan Maeder, PhD

10:20 AM – 12 PM

NIH Genome Editing Consortium Grant Recipients ICHAIR: Betty Poon, PhDBetty Poon, PhD

Introduction to NIH Genome Editing Consortium GrantsShengdar Tsai, PhD

A Novel Human T-cell Platform to Define Biological Adverse Effects of Genome EditingXue Wen, PhD

CRISPR-based in vivo gene editing in multiple target organsAravind Asokan, PhD

Evolving AAV for Efficient Neuromuscular Genome Editing

12 PM – 1 PM

Lunch

1 PM – 2 PM

Corporate Review I CHAIR: Paula Cannon, PhDShawdee Eshghi, PhD

Identification of high efficiency and high specificity guides for CRISPR-Cas9 engineering of allogeneic CAR-T cellsLeonela Amoasii, PhD

CRISPR/Cas9-Based Gene Editing for the Correction of Duchenne Muscular DystrophyDerek Jantz, PhD

Engineered Meganucleases for Gene and Cell TherapyCharlie Albright, PhD

Gene editing therapies for inherited retinal diseasesGiuseppe Ciaramella, PhD

Base Editing approaches to tackle serious genetic diseasesTony Ho, MD

Bringing Crispr to the Clinic

2 PM – 3 PM

NIH Genome Editing Consortium Grant Recipients II CHAIR: Betty Poon, PhDErik Sontheimer, PhD

Enhancing Genome Editing with Chemically Engineered Guides and New Cas9 OrthologsShaoqin Sarah Gong, PhD

Multifunctional CRISPR/Cas9 Delivery Nanoplatforms for Targeted Genome EditingZheng-Yi Chen, DPhil

Development of bioreducible lipid nanoparticles for the delivery of editing agents in the mammalian inner ear

Program



Program – continued

3 PM – 3:30 PM

Coffee break

3:30 PM – 5 PM

Junior Investigator Session CHAIR: Pablo Perez-Pinera, MD, PhDPietro Genovese, PhD

Advanced Genetic Engineering of Hematopoiesis for the Treatment of Inherited DiseasesBen Kleinstiver, PhD

Enhancing Genome Editing with Engineered CRISPR ProteinsThomas Gaj, PhD

Application of Genome Editing Technologies for the Treatment of Neurodegenerative DiseasesPrashant Mali, PhD

Therapeutic strategies via genome and transcriptome engineeringPinar Akcakaya, PhD

Evaluating in vivo off-targets of CRISPR-Cas gene editingJennifer Adair, PhD

Precision gene editing without electroporation in blood repopulating cells using a novel CRISPR nanoformulation

5 PM —6 PM

Corporate Review IICHAIR: Charles Gersbach, PhDEdward Rebar, PhD

Therapeutic Genome Editing and Gene Regulation Using Designed Zinc Finger ProteinsJulianne Smith, PhD

TALEN Gene Editing for “Off-the-Shelf” Allogeneic CAR T-Cell ProductsPhilip Gregory, DPhil

MegaTAL driven genome editing for enhanced CAR-T cell anti-tumor activityAndy Scharenberg, MD

Casebia therapeutics: driving gene editing innovationFred Chereau

Innovative promoterless nuclease-free genome editingSean Burns, PhD

Delivering on the therapeutic promise of CRISPR/Cas9



Co-ChairsPaula M. Cannon, PhDUniversity of Southern CaliforniaLos Angeles, CA

Charles Gersbach, PhDDuke UniversityDurham, North Carolina

Committee Members Angelo Lombardo, PhDSan Raffaele Telethon Institute for Gene TherapyMilan, Italy

Pablo Perez Pinera, MD, PhDUniversity of Illinois at Urbana-ChampaignUrbana, IL

David J. Rawlings, MDUniversity of WashingtonSeattle, WA

Morgan Maeder, PhDEditas Medicine Cambridge, MA

Speaker Listing

Co-Chairs Faculty Listing

Jennifer Adair, PhDFred Hutchinson Cancer Research CenterSeattle, WA

Pinar Akcakaya, PhDAstraZenecaGothenburg, Sweden

Charlie Albright, PhDEditas Medicine Cambridge, MA

Leonela Amoasii, PhDUT Southwestern Medical CenterDallas, TX

Aravind Asokan, PhDDuke University School of MedicineDurham, NC

Sean Burns, MDIntellia TherapeuticsCambridge, MA

Zheng-Yi Chen, DPhilMassachusetts Eye & Ear InfirmaryBoston, MA

Fred Chereau, MBALogicBio TherapeuticsCambridge, MA

Giuseppe Ciaramella, PhDBeam TherapeuticsCambridge, MA

Shawdee Eshghi, PhDCaribou BiosciencesBerkeley, CA

Thomas Gaj, PhDUniversity of IllinoisUrbana, IL

Pietro Genovese, PhDSan Raffaele Telethon Institute for Gene TherapyMilan, Italy

Shaoqin Sarah Gong, PhD Wisconsin Institutes for DiscoveryMadison, WI

Philip Gregory, DPhilBluebird BioCambridge, MA

Tony Ho, MDCRISPR TherapeuticsCambridge, Massachusetts

Derek Jantz, PhDPrecision BioSciences, Inc. Durham, NC

Benjamin Kleinstiver, PhDMassachusetts General HospitalBoston, MA

Prashant Mali, PhDUC San Diego Jacobs School of EngineeringLa Jolla, CA

Betty Poon, PhDNIH/NIAIDBethesda, MD

Edward Rebar, PhDSangamo BiosciencesRichmond, CA

Andy Scharenberg, MDCasebia TherapeuticsCambridge, MA

Julianne Smith, PhDCellectisNew York, NY

Erik Sontheimer, PhDUniversity of Massachusetts Medical SchoolWorcester, MA

Shengdar Tsai, PhDSt. Jude Children’s Research HospitalMemphis, TN

Wen Xue, PhDUniversity of Massachusetts Medical SchoolWorcester, MA



Speaker BiosJennifer Adair, PhDDr. Jennifer Adair received a Bachelor’s degree in Chemistry from Youngstown State University in Ohio and a Ph.D. in Genetics and Cell Biology from Washington State University. After a post-doctoral fellowship at the National Institute for Environmental Health Sciences, Jen joined Fred

Hutch as a Research Associate in the laboratory of Hans-Peter Kiem in 2008 to translate blood cell gene therapy for the treatment and prevention of malignant, genetic and infectious diseases. She joined the Fred Hutch faculty in 2014. The primary focus of Jen’s lab is in developing technologies that make gene therapy available beyond the high-tech, sterile labs that exist in just a handful of clinics in elite, developed countries. This includes automating gene modified, patient-specific cell manufacturing and developing novel technologies for genetic modification and gene editing in patients.

Pinar Akcakaya, PhDDr Pinar Akcakaya is a senior research scientist in the Discovery Sciences department at AstraZeneca. Pinar obtained her PhD at the Karolinska Institute, Stockholm, where she investigated the role of microRNAs in imatinib resistance in gastrointestinal stromal tumor cells

and patients.

Following that, she joined the AstraZeneca postdoctoral research program in Marcello Maresca`s team in Gothenburg, Sweden. As a postdoctoral scientist, Pinar worked with Keith Joung`s team in Harvard University and established an approach to validate the genome-wide precision of CRISPR‐Cas9 mediated gene engineering systems in vivo. This work demonstrated for the first time, that appropriately designed gRNAs can direct efficient in vivo editing without inducing detectable off-target mutations, a milestone for the clinical progression of CRISPR medicines. Following the completion of her postdoctoral work, Pinar joined the Precise Genome Editing team in AstraZeneca Gothenburg, where she is currently using CRISPR genome editing technologies to create disease models, to validate new drug targets and to generate CRISPR medicines.

Faculty Bios

Charlie Albright, PhDCharlie is Chief Scientific Officer of Editas Medicine. He joined Editas Medicine in August 2016 and is responsible for leading the team that is translating the promise of genome editing into a broad class of transformative genomic medicines.

Charlie brings more than 25 years of life sciences industry and academic leadership experience to this role. Throughout his career, Charlie has led discovery programs that advanced investigational medicines into clinical development in a wide range of therapeutic areas, including neurodegeneration, pain, psychiatry, oncology and inflammation.

Before joining Editas Medicine, Charlie spent 14 years at Bristol-Myers Squibb in scientific leadership roles in neuroscience biology. Most recently, he served as Vice President of Genetically-defined Diseases and Genomics. Prior to Bristol-Myers Squibb, Charlie held positions at Incyte Corporation and DuPont Pharmaceuticals and was an assistant professor of biochemistry at Vanderbilt University.

Charlie earned a B.S. in chemical engineering and a Ph.D. in biology from the Massachusetts Institute of Technology (MIT). He was a postdoctoral fellow in the laboratory of Professor Robert Weinberg at the Whitehead Institute for Biomedical Research at MIT.

Leonela Amoasii, PhDDr. Leonela Amoasii is a postdoctoral researcher at UTSW in Dr. Olson Laboratory. Dr. Amoasii pursued her graduate studies at University of Strasbourg in France. During her doctoral studies at the “Institut de Biologie Moleculaire et Cellulaire” (IGBMC), she worked on understanding mechanistic basis of

myotubular centronuclear myopathy in vivo using adeno associated virus (AAV). Dr. Amoasii has been involved in an important discovery of new therapeutic approach for correction of Duchenne muscular dystrophy using CRISPR/Cas9 genomic editing. Dr. Amoasii pursued the in vivo optimization of the CRISPR/Cas9 genomic editing, her recent ongoing work reveals promising results for translation of the genome editing technology to human patients. Dr. Amoasii is director of gene editing research at Exonics Therapeutics and will play a key role in leading the scientific efforts of the company.



Speaker Bios – continuedAravind Asokan, PhDAravind Asokan, PhD is a Professor at Duke University School of Medicine. His lab is focused on engineering viral elements for gene therapy, gene regulation and gene editing. One arm utilizes structural biology, protein engineering and directed evolution to generate viral vectors with

novel function(s) to treat genetic disease. The other arm is focused on engineering RNA structure & biology to control gene expression. In the past decade, the Asokan lab has made significant contributions towards understanding and re-engineering the biology of Adeno-Associated Viruses (AAV). This approach has yielded several recombinant AAV vector platforms being evaluated in preclinical and clinical gene transfer studies to treat genetic disorders such as muscular dystrophy, cardiac failure and neuropathies. In parallel, Dr. Asokan and his team are developing new tools to control mRNA stability and translation to achieve precise spatiotemporal control of gene expression. The overall mission of the Asokan lab is to translate these innovative platforms into transformative, new medicines.

Sean Burns, MDDr. Burns joined Intellia as the Senior Director of Hematology and New Therapeutic Areas in July 2015 from his prior role as a physician-scientist at Massachusetts General Hospital (MGH) and the Broad Institute of Harvard and MIT. Sean received his B.S. in Electrical Engineering from Cornell

University, and his M.D. with Honors from the New York University School of Medicine. He completed a residency in Internal Medicine at Beth Israel Deaconess Medical Center in Boston, followed by a fellowship in Endocrinology, Diabetes and Metabolism at Massachusetts General Hospital. He then served as an Assistant in Medicine within the Endocrine Division at MGH, where he provided care for patients with diabetes and other metabolic disorders. In the laboratory, he was a fellow within the Department of Molecular Biology, applying human genetic discoveries and genome engineering to identify and validate potential drug targets. He brings extensive experience with CRISPR/Cas9 to his role as Senior Director of Hematology and New Therapeutic Areas at Intellia Therapeutics, where he leads the effort to identify novel applications of CRISPR to genetic disease.

Paula Cannon, PhDPaula Cannon is a professor at the Keck School of Medicine of the University of Southern California. She studies the genetic modification of hematopoietic stem cells (HSC) to treat HIV infection and genetic diseases, where she is especially known for her work using gene editing

technologies such as zinc finger nucleases (ZFNs) and CRISPR/Cas9.

Her group has made a number of technical advances that have accelerated their application, including the demonstration that ZFNs could precisely knock out the CCR5 gene in HSC, and thereby produce an anti-HIV effect. These findings led to funding from the California Institute of Regenerative Medicine to develop a clinical trial in HIV-infected individuals. More recently, she has described a platform using AAV vectors in HSC that allows broader applications of gene editing, beyond gene knock-out. Finally, she is using humanized mouse models in several ongoing collaborations, including the evaluation of therapies for neurological diseases.

Zheng-Yi Chen, DPhilDr. Zheng-Yi Chen, is currently an associate professor of the Department of Otolaryngology, Harvard Medical School and Massachusetts Eye & ear Infirmary. His laboratory is focused on the development new treatment for human deafness. He obtained BSc from Sichuan University in China

and his DPhil. in human genetics from Oxford University, UK. He did his postdoctoral fellowship at the Massachusetts General Hospital/Harvard Medical School.

The work of his laboratory includes inner ear regeneration in mature mammalian inner ear, viral based and CRISPR genome editing based gene therapy to treat diverse forms of hearing loss including genetic hearing loss, age-related and environmental-related hearing loss. His laboratory has been involved in identification and characterization of numerous genetic deafness genes and in creation of large animal pig models for human genetic hearing loss. One of the main focuses of his laboratory is to develop novel delivery vehicles for editing agents in the inner ear for therapeutic application in human patients.

Fred Chereau, MBAFrederic Chereau has served as President and CEO of LogicBio Therapeutics since April 2016. Mr. Chereau is a global healthcare executive with more than 25 years of experience leading groundbreaking, high-growth biotherapeutics companies, moving products from proof-of-

concept pipeline, and through clinical trials toward successful commercialization. Before joining LogicBio, Mr. Chereau held various general management, business development and marketing positions at Genzyme, Pervasis Therapeutics, Shire and aTyr Pharma. He holds a bachelor’s degree in physics from Paris University, a master’s degree from ESC La Rochelle, and an M.B.A. from INSEAD.



Speaker Bios – continuedGiuseppe Ciaramella, PhDDr. Giuseppe Ciaramella is the Chief Scientific Officer of Beam Therapeutics. Dr. Ciaramella has more than 20 years of drug discovery experience at Moderna, AstraZeneca, Boehringer Ingelheim, Pfizer and Merck. Throughout his career he has held several leadership roles, with a particular

focus in the fields of antivirals, immunology and biotherapeutics. Prior to joining Beam Therapeutics, Dr. Ciaramella was the CSO of the Infectious Diseases division of Moderna Therapeutics, where he was instrumental in generating some of the first mRNA vaccines to be dosed in humans, several of which are progressing through clinical studies. At Boehringer Ingelheim, he served as Vice President and Head of Collaborative Research where he had responsibility for external R&D and was a member of the Research Leadership Team. Prior to Boehringer Ingelheim, he spent 14 years at Pfizer in the UK where he held several Discovery leadership positions, including head of Biotherapeutics, head of Antivirals and head of Lead Discovery.

He has contributed to several clinical candidates, both small molecule and biologics and to the anti-HIV drug Maraviroc (Selzentry TM), which won the USA Prix Galien for Best Pharmaceutical in 2008. He is a member of the Infectious Diseases Society of America (IDSA) and of the American Society of Gene Therapy (ASGT). Dr. Ciaramella holds a Ph.D. in Biochemistry and Molecular Biology from University College London.

Shawdee Eshghi, PhDShawdee Eshghi, Ph.D., is a Senior Scientist in the Cell Engineering Group at Caribou Biosciences. At Caribou, Shawdee is a Project Lead for a novel allogeneic immune cell therapy product candidate and also heads immune cell assay development across the company. In addition to cell therapy, she

has broad experience in other applications of gene editing technology including high throughput screening and synthetic biology. Prior to joining Caribou, Shawdee developed bispecific therapeutics for regenerative medicine applications in heart and kidney. Shawdee was an NIH Ruth L. Kirschstein Postdoctoral Fellow at the University of California, Berkeley, and holds doctoral and undergraduate degrees from the Massachusetts Institute of Technology in Biological Engineering and Chemical Engineering. Her academic research interests were at the intersection of bioengineering and stem cell biology, in particular the effects of the cellular microenvironment on stem cell function.

Thomas Gaj, PhDThomas Gaj is an Assistant Professor in the Department of Bioengineering and the Institute of Genomic Biology at the University of Illinois. He received his Ph.D. in Chemistry from the Scripps Research Institute in La Jolla, CA and performed his postdoctoral research at the University of

California, Berkeley. His lab is focused on developing corrective gene therapies for neurodegenerative conditions.

Pietro Genovese, PhDDr. Pietro Genovese, Ph.D., is Project Leader of the Genome Editing Unit at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, Italy. Author of several high-profile publications, in the last 10 years he dedicates his efforts to develop genome editing tools that improve the safety and

efficacy of adoptive immunotherapy or promote safer applications of human stem cell gene therapy. He recently designed a novel strategy that allows site-specific integration and in situ gene correction in human long-term repopulating hematopoietic stem cells. His current efforts are aimed to move these advanced genetic engineering strategies towards an effective therapeutic treatment for inherited diseases. Dr. Genovese is co-inventor of more than 5 patents on gene editing technology, recipient of several awards, from which the Young Investigator Award from the ESGCT society (2016), and was invited to present his research at several internationals meetings.

Charles Gersbach, PhDDr. Charles A. Gersbach is the Rooney Family Associate Professor at Duke University in the Departments of Biomedical Engineering and Orthopaedic Surgery, an Investigator in the Duke Center for Genomic and Computational Biology, and Director of the Duke Center for Biomolecular

and Tissue Engineering. His research interests are in genome and epigenome editing, gene therapy, regenerative medicine, biomolecular and cellular engineering, synthetic biology, and genomics. Dr. Gersbach’s work has been recognized through awards including the NIH Director’s New Innovator Award, the NSF CAREER Award, the Outstanding New Investigator Award from the American Society of Gene and Cell Therapy, the Allen Distinguished Investigator award, and induction as a Fellow of the American Institute for Medical and Biological Engineering.



Shaoqin Sarah Gong, PhD Dr. Shaoqin Sarah Gong is a Vilas Distinguished Professor in the Department of Biomedical Engineering and the Wisconsin Institute for Discovery at the University of Wisconsin–Madison. She is also a member of the UW Carbone Cancer Center, Stem Cell and Regenerative Medicine

Center, and McPherson Eye Research Institute, and an affiliate faculty member in the Department of Chemistry and Department of Materials Science and Engineering. She received her BA and MS degrees from Tsinghua University and her PhD degree from the University of Michigan–Ann Arbor—all in Materials Science and Engineering. Currently, Dr. Gong’s research focuses on the design, synthesis/fabrication, and characterization of novel nanomaterials and biomaterials for various biomedical applications. Many of her on-going projects are multidisciplinary, bridging engineering with materials science, chemistry, pharmacy, and the life sciences. Dr. Gong is a fellow of the American Institute for Medical and Biological Engineering and an editorial board member for several journals including Biomaterials, Theranostics, Biofabrication, and Nanotheranostics. She also served as an Associate Editor for Biomaterials. Dr. Gong is a winner of a number of awards including the NSF CAREER Award and NIH Faculty Career Development Award.

Philip Gregory, DPhilPhilip D. Gregory, D. Phil., has served as Chief Scientific Officer of bluebird bio since June 2015. Prior to joining bluebird Philip was at Sangamo BioSciences, Inc., where he held multiple leadership positions, most recently serving as Chief Scientific Officer and Senior Vice President,

Research. Philip has led pioneering research in gene therapy and genome editing and was named by Thompson Reuters as one of “The World’s Most Influential Scientific Minds 2014” and “Most Cited Researchers 2015”. He served as a member of the Scientific Advisory Board of the Keystone Symposia from 2009 to 2015, and the ASGCT Advisory Council from 2016. Before joining the biotechnology industry Philip was at the University of Munich, Germany, where he studied the role of chromatin structure in gene regulation. Philip earned a D. Phil. in biochemistry from the University of Oxford (Keble College) and holds a B.Sc. in microbiology from the University of Sheffield.

Tony Ho, MDDr. Tony Ho is Executive Vice President, Head of Research and Development at CRISPR Therapeutics. He was previously Senior Vice President and Head of Oncology Integration and Innovation at AstraZeneca. During his tenure at AstraZeneca, he oversaw both development and

commercialization of the two key drugs – Lynparza, a first-in-class PARP inhibitor for ovarian cancer and Imfinizi (anti-PD-L1), AstraZeneca’s first immuno-oncology drug for bladder cancer. Prior to joining AstraZeneca, Tony was the Neurology and Ophthalmology Clinical Section Head at Merck Research Laboratories, Merck & Co., Inc. and led multiple development programs including the approval of Maxalt for pediatric migraine and Zioptan for glaucoma. Prior to joining Merck, Tony was the Co-Founder and Chief Scientific Officer of Neuronyx, Inc., a regenerative medicine company.

Tony completed his B.S. in Electrical Engineering at the University of California, Los Angeles, and received his M.D. from the Johns Hopkins University School of Medicine. After an internship in Internal Medicine at the Massachusetts General Hospital, Tony completed his residency and neurophysiology fellowship in the Department of Neurology at the Johns Hopkins Hospital. He was Assistant Professor at Johns Hopkins Hospital in the areas of neuropathy and neuromuscular diseases. Tony describing and elucidating the pathogenesis of a new disease called “acute motor axonal neuropathy (AMAN)”. He has published widely in several fields with over 80 papers. He is currently adjunct Associate Professor of Neurology at University of Pennsylvania and Assistant Professor (Part-time) of Neurology at Johns Hopkins University.

Derek Jantz, PhDDerek is a veteran of the genome editing industry. He was involved in early research with the zinc finger technology and has spent most of his research career designing proteins for genome editing applications. He has a B.A. in biology from the University of Colorado at Boulder and a PhD in

Biophysics from Johns Hopkins University School of Medicine. Dr. Jantz co-founded Precision BioSciences in 2006 after co-inventing a novel method for modifying the DNA-recognition properties of homing endonucleases. Since that time, he has served as VP of Scientific Development and Chief Scientific Officer at the company.

Speaker Bios – continued



Benjamin Kleinstiver, PhDBen Kleinstiver is an Assistant Investigator at the Massachusetts General Hospital (MGH) and Assistant Professor at Harvard Medical School (HMS). His research interests are broadly focused on developing and improving genome editing technologies for biomedical research and clinical

applications. The work in his laboratory is focused on using protein engineering methods to evolve genome editing nucleases that have enhanced properties. His research group is actively developing CRISPR enzymes with improved on-target activities, genome-wide specificities, and targeting ranges, and are also exploring the feasibility of applying these engineered technologies for the treatment of genetic diseases.

Prashant Mali, PhDPrashant Mali received a bachelor’s and master’s in Electrical Engineering from the Indian Institute of Technology Bombay, with a research focus on solid-state biosensors; and a doctorate in Biomedical Engineering at the Johns Hopkins University, with a research focus on human

pluripotent stem cell engineering. During his postdoctoral fellowship in the Department of Genetics at the Harvard Medical School, he pioneered the development of the CRISPR-Cas systems for eukaryotic genome engineering. For his graduate work he received the Siebel Scholar Award in 2011, and in 2014 he received the Burroughs Wellcome Career Award at the Scientific Interface. Mali joined the University of California, San Diego faculty in the Fall of 2014 as an assistant professor in the Department of Bioengineering. In 2016 he received the March of Dimes Basil O’Conner Scholar Award, the Kimmel Scholar Award, and was named a Kavli Frontiers of Science Fellow. In 2017 he received the Young Alumnus Achiever Award from the Indian Institute of Technology Bombay India. Research in his laboratory lies at the interface of basic science and technology development, with a core thrust in developing tools for engineering biology towards enabling gene and cell based human therapeutics.

Betty Poon, PhDDr. Betty Poon currently is a Program Officer in the Basic Sciences Program, Division of AIDS, at the National Institute of Allergy and Infectious Disease (NIAID), National Institutes of Health (NIH). In this role, she oversees an active scientific portfolio of grants, cooperative agreements including multi-

project programs and clinical trials, and contracts, with a focus on the development of cell and gene therapeutics for an HIV cure. She also is on the Program Leadership Team for the coordination and management of the NIH Common-fund supported Somatic Cell Genome Editing (SCGE) Consortium with the goal of accelerating the translation of genome editing technology into clinical applications. Dr. Poon has broad experience in biomedical research particularly in the areas of molecular biology, virology, and novel therapeutics development. She first came to NIAID as a Scientific Review Officer where she organized and lead scientific review meetings for various NIH biomedical and behavioral research programs including HIV immunopathogenesis and cure, vaccines, mucosal immunology, clinical and epidemiology research, and biodefense. Prior to joining NIAID, Dr. Poon was an Assistant Research Biologist at UCLA where she collaborated on the pre-clinical development of an inactivated HIV vaccine and gene therapy research targeted against HIV. She earned a Bachelor of Science degree in Molecular Biology from Princeton University, and her Ph.D. in Microbiology and Immunology from UCLA.

Edward Rebar, PhDEd Rebar, Ph.D., is Chief Technology Officer at Sangamo Therapeutics, which is focused on developing designed zinc finger proteins for therapeutic editing and regulation of targeted genes. In prior roles at Sangamo Ed has served as Vice President and Senior Director of Technology,

capacities in which he has directed the design and characterization of zinc finger proteins for therapeutic applications and also helped to establish Sangamo’s zinc finger protein design platform. Ed has coauthored over 60 publications relating to the development of customized DNA binding proteins and nucleases for genome editing, as well as numerous patents. Prior to joining Sangamo he was a post-doctoral fellow at the University of California, Berkeley. Ed earned his B.S. in Biochemistry from Rutgers University and his Ph.D. in Biophysics and Structural Biology from MIT.




Andy Scharenberg, MDAndrew M. Scharenberg, MD, is an Attending Physician at Seattle Children’s Hospital, a Professor in the Department of Pediatrics, and an Adjunct Professor in the Department of Immunology at the University of Washington School of Medicine in Seattle, from which he is currently on leave. He

presently serves as Chief Scientific Officer of Casebia Therapeutics in Cambridge, MA.

Prior to joining Casebia, Dr. Scharenberg co-directed (with David Rawlings M.D.) the Program in Cell and Gene Therapy at Seattle Children’s Research Institute, working to translate cell and gene therapies for the treatment of inherited immunologic and blood diseases. This work led to the development of a program in engineered regulatory T-cells, recently partnered with Casebia for the purpose of developing gene edited cell therapeutics for IPEX syndrome. Dr Scharenberg also is a past Chief Scientific Officer of Cellectis Therapeutics, where he initiated the development of an allogeneic CAR T-cell platform, and past co-founder of Pregenen Inc., a gene editing and cell-signalling technology company that was acquired by bluebird bio in 2014. He earned his medical degree from the University of North Carolina School of Medicine, and then completed residency training in pediatrics at the University of North Carolina Children’s Hospital and his fellowship in immunology at NIH and at the Division of Experimental Pathology, Beth Israel Hospital.

Julianne Smith, PhDJulianne Smith Ph.D. joined Cellectis in 2002 and has been working in the therapeutic division since its inception. As VP CART Development, she was involved in the development of Cellectis’ Universal CART (UCART) products overseeing product development and nonclinical strategy. She is

currently VP Translational Sciences and is leading the correlative studies for the UCART clinical trials. Dr. Smith has a Ph.D.in Genetics and Development from Columbia University and a B.A. in Biology from Johns Hopkins University

Erik Sontheimer, PhDErik Sontheimer, Ph.D., is Professor in the RNA Therapeutics Institute at the University of Massachusetts Medical School. He completed his Ph.D. at Yale in the laboratory of Joan Steitz and was a postdoctoral fellow with Joe Piccirilli at the University of Chicago. In 1999 Sontheimer joined

the faculty at Northwestern where he continued his work on the roles of RNA molecules in gene expression, including the study of CRISPR immune systems in bacteria. Among other advances, in 2008 his group demonstrated that CRISPR systems target DNA molecules directly, and they became the first to recognize and articulate CRISPR’s potential for genome engineering. He has received a CAREER Award from the NSF, a New Investigator Award from the Burroughs Wellcome Fund, a Distinguished Teaching Award from Northwestern, the Nestlé Award from the American Society for Microbiology, the Mid-Career Award from the RNA Society, and election to the American Academy of Microbiology. In 2014 he co-founded Intellia Therapeutics, Inc. for the development of clinical applications of CRISPR gene editing. That same year he also moved to the RNA Therapeutics Institute at UMass Medical School, where he is continuing his research on the biological roles and technological applications of RNA-based systems.

Shengdar Tsai, PhDDr. Tsai is an Assistant Member in the Department of Hematology at St. Jude Children’s Research Hospital. His lab focuses on genome editing technologies for therapeutics, with a special interest in editing human HSCs for treatment of hemoglobinopathies and T-cells for cancer

immunotherapy. Previously, he has developed methods for high-throughput genome editing with TALENs, and for defining and improving the genome-wide specificity of CRISPR-Cas nucleases. Dr. Tsai completed a postdoctoral fellowship at Massachusetts General Hospital & Harvard Medical School, Ph.D. in Functional Genomics and M.S. in Bioinformatics from North Carolina State University, and B.S. from the University of Michigan.

Wen Xue, PhDWen Xue is an Assistant Professor in the RNA Therapeutics Institute at University of Massachusetts Medical School. His research focuses on cancer genetics using mouse models and study of genetic diseases using CRISPR genome editing. He has a strong background in

RNA biology and gene therapy. He did Ph.D. training with Dr. Scott Lowe at the Cold Spring Harbor labs and Stony Brook University (2005-2009) and postdoctoral training under the mentorship of Dr. Tyler Jacks at MIT (2009-2014). He has received awards including the NIH Director’s New Innovator Award, Scientific Merit Award of the Lung Cancer Research Foundation, and the American Cancer Society Research Scholars Grant. He is on the editorial board of Human Gene Therapy and FASEB




Disclosure of Relevant Financial Relationships

Disclosure of Relevant Financial Relationships

Pinar Akcakaya, PhD AstraZeneca AB: salary and shares

Charlie Albright, PhD Editas Medicine: salary, stock options Jnana: compensation and stock options

Leonela Amoasii, PhD Exonics Therapeutics: salary

Aravind Asokan, PhD StrideBio, Inc.: Equity, consulting fee, royalty Gemini Therapeutics, Inc.: Consulting fee UCB Pharma: Consulting fee Casebia Therapeutics, Inc.: Consulting fee TorqueBio, LLC: Equity

Sean Burns, MD Intellia Therapeutics: salary, equity

Zheng-Yi Chen, DPhil Decibel Therapeutics Inc.: consulting fee

Fred Chereau, MBA LogicBio Therapeutics: salary, equity Dynacure: equity, consulting fees

Giuseppe Ciaramella, PhD Beam Therapeutics: salary, shares

Shawdee Eshghi, PhD Caribou Biosciences: salary stock options Silver Creek Pharmaceuticals: salary, stock options

Thomas Gaj, PhD 4D Molecular Therapeutics: consulting fee VL58: consulting fee

Pietro Genovese, PhD Patent applications on HSC gene editing protocols

Philip Gregory, DPhil bluebird bio: salary, stock options

Tony Ho, MD CRISPR Therapeutics: salary, stock

Derek Jantz, PhD Precision BioSciences, Inc.: salary and equity

Morgan Maeder, PhD Editas Medicine: stock holder

Prashant Mali, PhD Pretzel Therapeutics equity and consulting fees Shape Therapeutics: equity and consulting fees Engine Biosciences: equity and consulting fees Seven Therapeutics: equity and consulting fees Navega Therapeutics: equity and consulting fees

Pablo Perez Pinera, MD, PhD Editas Medicine, Inc. and Elements Genomics: Royalties

Edward Rebar, PhD Sangamo Biosciences: salary, stock based compensation

Julianne Smith, PhD Cellectis: salary

Erik Sontheimer, PhD Intellia Therapeutics: ownership interest, consulting fees

Shengdar Tsai, PhD Vertex Pharmaceuticals: honoraria



Notes

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Gene Editing Workshop...April 28, 2019 Washington, D.C. 5 Washington Hilton Gene Editing Workshop...

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