Gene Editing Workshop

Gene Editing Workshop 21st Annual Meeting ASGCT CHICAGO, ILLINOIS 2018 MAY 16-19 May 15, 2018 Hilton Chicago Continental C Chicago Gene Editing Workshop Table of Contents Workshop Supporters . p 2 Committee Listing . p 3 Faculty Listing . p 3 Faculty Bios . p 4 Disclosure of Relevant Financial Relationships . p 8 Program Schedule . p 9 Notes . p 11 American Society of Gene & Cell Therapy 1 May 15, 2018 Chicago Hilton Chicago Gene Editing Workshop Gene Editing Workshop Supporters The American Society of Gene & Cell Therapy is honored to acknowledge the following organizations for their support of the Gene Editing Workshop: American Society of Gene & Cell Therapy 2 May 15, 2018 Chicago Hilton Chicago Gene Editing Workshop Committee Listing Co-Chairs J. Keith Joung, MD, PhD Matthew H. Porteus, MD, PhD Massachusetts General Hospital Stanford University School of Medicine Charlestown, MA Stanford, CA Members Paula M. Cannon, PhD Toni Cathomen, PhD Charles A. Gersbach, PhD University of Southern California Medical Center - University of Freiburg Duke University Los Angeles, CA Freiburg, Germany Durham, NC Faculty Listing Omar Abudayyeh Beverly L. Davidson, PhD J. Keith Joung, MD, PhD Broad Institute of MIT and Harvard Children’s Hospital of Philadelphia Massachusetts General Hospital Cambridge, MA Philadelphia, PA Charlestown, MA Charlie Albright, PhD Suk See De Ravin, MD, PhD Alexis C. Komor, PhD Editas Medicine National Institutes of Health, NIAID UC San Diego Cambridge, MA Bethesda, MD La Jolla, CA Leonela Amoasii, PhD Daniel Dever, PhD Vikram Pattanayak, MD, PhD UTSW Medical Center Stanford University Medical Center Massachusetts General Hospital Dallas, TX Stanford, CA Boston, MA Thomas Barnes, PhD Justin Eyquem, PhD Krishanu Saha, PhD Intellia Therapeutics Memorial Sloan Kettering Cancer Center University of Wisconsin-Madison Cambridge, MA New York, NY Madison, WI Mark A. Behlke, MD, PhD Nicole Gaudelli, PhD Julianne Smith, PhD Integrated DNA Technologies, Inc . Beam Therapeutics Cellectis Coralville, IA Boston, MA New York, NY Paula M. Cannon, PhD Charles A. Gersbach, PhD James M. Wilson, MD, PhD University of Southern California Duke University University of Pennsylvania Los Angeles, CA Durham, NC Philadelphia, PA Toni Cathomen, PhD Ayal Hendel, PhD Medical Center - University of Freiburg Bar Ilan University Freiburg, Germany Ramat-Gan, Israel Tirtha Chakraborty, PhD Michael C. Holmes, PhD CRISPR Therapeutics Sangamo BioSciences, Inc Cambridge, MA Richmond, CA American Society of Gene & Cell Therapy 3 May 15, 2018 Chicago Hilton Chicago Gene Editing Workshop Faculty Bios Omar Abudayyeh scientific visions into successful businesses for both startups and Omar O . Abudayyeh is an MD–PhD . student in the Harvard– established organizations . Massachusetts Institute of Technology (MIT) Health Sciences and Barnes has wide-ranging knowledge of biological systems through Technology program . In the laboratory of Feng Zhang at the Broad his work across diverse platforms, including genomics and gene Institute of MIT and Harvard, his doctoral research focuses on the discovery, small molecule drug repositioning, and protein discovery and characterization of novel CRISPR proteins, such as engineering . Previously, as vice president of discovery at Eleven Cpf1, C2c1, and Cas13a/C2c2 in bacteria for the purpose of Biotherapeutics, Tom led his team in creating a novel chimeric expanding the genome-editing toolbox and studying mammalian cytokine antagonist, as well as two novel technologies . As senior biology . His recent work has focused on using the RNA-targeting vice president and site head of GeneLogic’s drug repositioning CRISPR system Cas13 for building a transcriptome engineering division, he oversaw technology platforms in metabolomics, toolbox for applications in diagnostics and therapeutics . In 2012, toxicogenomics, gene expression informatics, and the genetics of Abudayyeh graduated with degrees in Mechanical Engineering drug metabolism and transport . Barnes received his PhD . from the and Biological Engineering from MIT . University of Cambridge, and completed research fellowships at Charlie Albright, PhD Harvard Medical School and McGill University . Charlie Albright joined Editas Medicine as Chief Scientific Officer in Mark A. Behlke, MD, PhD August 2016 . He brings more than 25 years of life sciences industry As the chief scientific officer, Dr . Mark A . Behlke has directed and academic leadership experience, most recently serving as vice research activities at IDT since joining the company in 1995 with a president of genetically defined diseases and genomics at Bristol- focus on novel molecular biology applications of oligonucleotide- Myers Squibb (BMS) . Over his career, Albright has led discovery based technologies . In addition, Behlke is also a scientific co- programs that advanced investigational medicines into clinical founder of Dicerna Pharmaceuticals, located in Boston . Before development in a wide range of therapeutic areas, including joining IDT, Behlke was a Physician Postdoctoral Fellow of the neurodegeneration, pain, psychiatry, oncology, and inflammation . Howard Hughes Medical Institute at the Whitehead Institute, MIT . Prior to his position as vice president of genetically defined He was a resident physician in internal medicine at Brigham and diseases and genomics, he held multiple scientific leadership roles Women’s Hospital, Boston . Behlke received his MD and PhD in neuroscience biology at BMS . Previously, he held positions at degrees from Washington University, St . Louis in 1988 and his B .S . Incyte Corporation and DuPont Pharmaceuticals and was an degree from the Massachusetts Institute of Technology in 1981 . assistant professor of biochemistry at Vanderbilt University . Charlie Behlke is an inventor on more than 45 issued US patents, has received a Bachelor of Science in Chemical Engineering and a PhD . numerous pending patent applications, and is an author on over in biology from the Massachusetts Institute of Technology (MIT) . 125 scientific publications and book chapters . He is a recognized He was a postdoctoral fellow in the laboratory of Professor Robert expert on oligonucleotide-based technologies . Weinberg at the Whitehead Institute for Biomedical Research at MIT . Paula M. Cannon, PhD Paula Cannon, PhD . is a distinguished professor in the Keck School Leonela Amoasii, PhD of Medicine of the University of Southern California in Los Angeles . Leonela Amoasii works in the laboratory of Dr . Eric Olson at the She studies genome engineering in hematopoietic stem cells, with University of Texas Southwestern Medical Center in Dallas . Amoasii an emphasis on developing therapies for HIV/AIDS . Cannon earned pursued her graduate studies at the Institut de Genetique Biologie her PhD . from the University of Liverpool and did post-doctoral Moléculaire et Cellulaire (IGBMC) at the University of Strasbourg in training at Harvard and Oxford Universities . In 2010, her team were France . During her doctoral studies, she uncovered the the first to show that genome engineering could be used to knock- mechanistic basis of myotubular centronuclear myopathy and out the CCR5 gene in human hematopoietic stem cells . The acquired expertise in the use of adeno-associated virus (AAV) for discovery has now led to an ongoing clinical trial in HIV-infected manipulation of gene expression in muscle . Over the last years, individuals . She continues to develop new applications for Amoasii has been involved in developing a new therapeutic genome engineering, with a goal of applying this technology to approach for correction of Duchenne muscular dystrophy using treat infectious and genetic diseases of the blood and immune CRISPR/Cas9 genomic editing . Amoasii pursued the in vivo systems . optimization of the CRISPR/Cas9 genomic editing, her recent work reveals promising results for translation of the genome editing Toni Cathomen, PhD technology to human patients . Toni Cathomen is professor of cell and gene therapy and director of the Institute for Transfusion Medicine and Gene Therapy at the Thomas Barnes, PhD Medical Center of the University of Freiburg, Germany . After Tom Barnes has led platform-based research and drug discovery receiving his PhD . from the University of Zurich, Switzerland, he teams for over 20 years, and is responsible for extending the reach was a postdoctoral fellow at the Salk Institute in San Diego, of Intellia’s CRISPR platform into new areas . A veteran entrepreneur assistant professor of Molecular Virology at Charité Medical School who has helped launch several companies, he has turned creative in Berlin, and associate professor of experimental hematology at American Society of Gene & Cell Therapy 4 May 15, 2018 Chicago Hilton Chicago Gene Editing Workshop Faculty Bios – continued Hannover Medical School . His research activities focus on the of the National Ataxia Foundation, Davidson is a co-founder of development of immune cell therapies based on induced Spark Therapeutics, Inc ., and serves on the advisory boards of pluripotent stem cells (iPSCs); the improvement of the Sarepta Therapeutics and Intellia Therapeutics . effectiveness and safety of designer nucleases (CRISPR-Cas and TALEN) for targeted genome editing; and the development and Suk See De Ravin, MD, PhD manufacturing of genome edited stem cell and immune cell Suk See De Ravin completed her pediatric specialty training, preparations for the treatment of HIV infection, primary followed by research focusing on the

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