Rob has been a leader in adeno-associated virus (AAV) research for over 30 years, beginning as a post-doctoral fellow at Cornell University Medical Center (now Weill), where he discovered a common integration site for AAV DNA in human chromosome 19, which he designated AAVS1 locus. He spent most of his career in the Intramural Research Program at the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) as a tenure track inves tigator then as a tenured senior investigator and Head of the Laboratory of Molecular Biology and Gene Therapy. While at the NIH, Rob's laboratory invented and developed a scalable recombinant adeno-associated virus (rAAV) production process in Sf9 cells which was licensed by several companies and was used to produce Glybera™, the first rAAV product granted regulatory agency approval for sale. The research at the NIH resulted in the discovery of an AAV replicative product that has been described as closed-ended linear duplex DNA. Rob served as vice president of virology and gene therapy at Voyager Therapeutics from 2014 to 2016. Rob earned his Bachelor of Arts in biology from the University of California, Santa Cruz and his doctorate from Rutgers University and the University of Medicine and Dentistry of New Jersey (now Robert Wood Johnson Medical School) in microbiology.

Mark was senior vice president of pharmaceutical sciences and rare disease franchise head at bluebird bio Inc. from 2012 until 2016. Previously, he was senior director of research and development and site head in Cambridge, Massachusetts for Baxter Healthcare Corporation from 2010 to 2012. Prior to 2010, Mark worked at Archemix, Momenta Pharmaceuticals, Millennium Pharmaceuticals, and DuPont Pharmaceuticals. His experience spans from development through commercialization including the first commercial launch for a bioequivalent (generic Lovenox™), the ANDA submission for generic Copaxone™, the sNDA for Integrilin™, and the BLA for Zynteglo™ as well as numerous INDs across small-molecules, biologics, peptides, biologics, complex mixture, and gene therapy products. Mark holds a doctorate in chemical engineering and a Master of Science in chemical engineering practice from the Massachusetts Institute of Technology, and obtained his Bachelor of Science in chemical engineering from The Cooper Union.

Adrian Thrasher is professor of pediatric immunology and Wellcome Trust Principal Research Fellow at the University College London Great Ormond Street Institute of Child Health (UCL GOSICH), and Honorary Consultant Pediatric Immunologist at Great Ormond Street Hospital for Children National Health Service Foundation Trust (GOSH). He is the program head of Infection, Immunity and Inflammation at UCL GOSICH and has a longstanding research and clinical interest in the development and application of gene therapy. He is director of the Clinical Gene Therapy Program, and theme leader of the gene stem and cellular therapies theme of the Biomedical Research Centre, at UCL GOSICH/GOSH. Adrian is principal investigator on several clinical trials for immunodeficiency and is director of the clinical gene therapy GMP facility, managing a team of trial coordinators, clinical scientists, and quality systems personnel. His clinical interests are the diagnosis and treatment of patients with primary immunodeficiency. His specialist interests are in the Wiskott-Aldrich Syndrome (WAS), disorders of innate immunity, and autoimmune lymphoproliferative syndrome. His team at UCL GOSICH/GOSH is conducting trials of somatic gene therapy for various forms of PID including X-linked severe combined immunodeficiency (SCID-X1), Chronic granulomatous disease (CGD), Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID), and WAS. Research interests include the pathophysiology of primary immunodeficiency syndromes especially WAS, the actin cytoskeleton in haematopoietic cells, the development of somatic gene therapy, and thymus transplantation. Adrian qualified in medicine in 1986 at St. George’s Hospital Medical School, University of London. He earned his doctorate in 1995 at University College London.

Richard Thompson is the Professor of Molecular Hepatology at King’s College London and honorary consultant pediatric hepatologist at King’s College Hospital, London. He specializes in pediatric liver disease and is particularly interested in genetic liver disease in both children and adults. Through worldwide collaborations, Richard’s lab continues to identify new causes of genetic liver disease. Additionally, Richard served as the clinical lead for genomics and the 100,000 genome project at King’s College Hospital, and is the clinical lead for a diagnostic laboratory specializing in liver and gastrointestinal disease. He has been the principal investigator on several clinical trials Richard has published over 50 peer reviewed articles, as well as several book chapters and editorials. He has been an invited speaker at numerous conferences worldwide. Richard received his undergraduate, medical and research degrees from the University of Oxford.

Kevin Fitzgerald is senior vice president, head of research and translational science at Alnylam Pharmaceuticals in Cambridge, MA. He has over 19 years of drug discovery experience. He joined Alnylam in 2005 as an associate director of research after spending 7 years at Bristol-Myers Squibb. He has served in roles of increasing responsibility and leadership since that time. His achievements at Alnylam include leadership of the company's RNAi delivery efforts, resulting in two clinically validated modes of siRNA delivery, and the development of Alnylam’s disease pipeline. He is an inventor on over 50 patents including the majority of Alnylam’s pipeline programs, and the author of over 40 papers including many in prestigious journal’s such as Nature, Cell, and NEJM. He has led multiple programs — including Alnylam’s Inclisiran program — from discovery through pre-clinical development, regulatory submissions, and early clinical development. Kevin received his Bachelor of Science in genetics from Cornell University and his doctorate in molecular biology from Princeton University. He completed his post-doctoral fellowship in oncology at Harvard Medical School.

Katherine Fitzgerald is a professor of medicine and director of the Program in Innate Immunity within the Division of Infectious Disease and Immunology at the University of Massachusetts Medical School, where she has worked since 2001. At the University, she serves as principal investigator of the Fitzgerald Lab, working with post-doctoral fellows and graduate students to understand how dysregulation of innate immunity plays a central role in the pathogenesis of infectious, inflammatory and autoimmune disease. Dr. Fitzgerald has received numerous awards for her research and has been named a Thomson Reuters Highly Cited Researcher multiple times, recognizing her as being among the most frequently cited researchers in the field of immunology. Kate received her Bachelor of Science in biochemistry from the University of Cork, Ireland and her doctorate in biochemistry from Trinity College Dublin, Ireland.

Steven Dowdy is a professor of cellular and molecular medicine at the University of California San Diego School of Medicine, where his research has focused on the delivery of novel therapeutics, especially RNAi therapies, into cells. He previously served as an investigator at the Howard Hughes Medical Institute and an assistant professor of pathology and medicine at the Washington University School of Medicine in St. Louis. In addition to Generation Bio, he serves on the Scientific Advisory Boards of Deep Genomics and NeuBase Therapeutics and is a member of the Board of Directors of the Oligonucleotide Therapeutics Society. He is a coauthor of more than 100 peer-reviewed publications in cell and molecular medicine. Dr. Dowdy earned his Ph.D. in microbiology and molecular genetics at the University of California School of Medicine and holds a B.S. with honors from the School of Biological Sciences at the University of California Irvine. He conducted postdoctoral research with Dr. Robert Weinberg at the Whitehead Institute for Biomedical Research at the Massachusetts Institute of Technology.

Andy Scharenberg is chief scientific officer at Casebia. Before joining Casebia, Scharenberg was an attending physician at Seattle Children's Hospital and principal investigator in the Center for Immunity and Immunotherapies at Seattle Children's Research Institute. In addition, he served as co-director of the Program for Cell and Gene Therapy at Seattle Children's Hospital, a professor in the Department of Pediatrics and adjunct professor in the Department of Immunology at the University of Washington School of Medicine, and a member of the Transplantation Biology Consortium Program at Fred Hutchinson Cancer Research Center. As chief scientific officer at Cellectis, Scharenberg helped implement a gene-editing partnership with Pfizer. Andrew earned his Bachelor of Science in biochemistry from Indiana University and his medical degree from the University of North Carolina School of Medicine.

Catherine was appointed chief development officer of Obsidian Therapeutics in 2019. Previously, she was entrepreneur-in-residence at Atlas Ventures, serving as chief medical officer of both Dyne Therapeutics and Disarm Therapeutics. She also spent time as chief medical officer of Sarepta Therapeutics. Prior to that, Catherine served as vice president, clinical development and regulatory affairs at Regeneron Pharmaceuticals, initially as head, pain therapeutic area, and subsequently as head, clinical project management and operations. From 2003 to 2015, she held senior leadership roles at Amgen including vice president, global development, leading the neuroscience, nephrology and bone therapeutic areas. Dr. Stehman-Breen earned her M.D. from the University of Chicago in 1990. She conducted her residency and fellowship training at the University of Washington, where she also received a Master of Science degree in Epidemiology in 1996. Dr. Stehman-Breen spent six years as a faculty member in the Division of Nephrology at the University of Washington from 1997 to 2003, where her primary responsibilities included managing the Clinical Research Training Program and the development of the Epidemiology and Clinical Trials Research Program. Her research focused on bone and cardiovascular disease in kidney disease patients. Dr. Stehman-Breen was also active in a variety of national programs including serving as the Chair for USRDS NIDDK External Expert Panel and a reviewer for several journals.

Charlie has more than 30 years of biopharmaceutical industry experience and is currently a board member and strategic advisor for multiple biotechnology companies. He was most recently the president and chief executive officer (CEO) of Aurinia Pharmaceuticals, a clinical stage pharmaceutical company focused on the global lupus nephritis market. Prior to his tenure with Aurinia Pharmaceuticals, Charlie served as the vice president and chief financial officer (CFO) of ViroPharma, during which time the company grew into a global biopharmaceutical business with $500 million in annual revenues until it was acquired by Shire PLC for $4.2 billion. Before joining ViroPharma, he was executive vice president and CFO, as well as interim Co-CEO, for Endo Pharmaceuticals, a specialty pharmaceutical company with $1.3 billion in revenues. He previously held finance and operational positions of increasing responsibility at Biovail Pharmaceuticals, Breakaway Technologies, Pharmacia, Novartis and Bristol-Myers Squibb. Charlie currently serves as a member of the board of directors for Blueprint Medicines, Nabriva Therapeutics, Viking Therapeutics, Psioxus Therapeutics and Orchard Therapeutics with previous board appointments including Vitae Pharmaceuticals, BIND Therapeutics, Aurinia Pharmaceuticals and Idenix Pharmaceuticals, among others. He holds a Master of Business Administration from Rutgers University and a Bachelor of Science from Saint Joseph's University.

Anthony Quinn is president and chief executive officer at Aeglea BioTherapeutics. Previously, Dr. Quinn served as executive vice president, head of research and development, and chief medical officer at Synageva Biopharma Corp., until the company’s acquisition by Alexion Pharmaceuticals in 2015. During his tenure at Synageva, he played a key role in securing the European and U.S. approvals of Kanuma™ for Lysosomal Acid Lipase Deficiency and in building the company’s research and development organization and rare disease drug pipeline. Prior to his role at Synageva, Anthony served as worldwide head of clinical research and exploratory development for inflammatory diseases at Roche. Previously, he was a professor of dermatology at Barts and The London School of Medicine. Anthony received his Bachelor of Medical Science and his M.B Ch.B from the University of Dundee, and a Ph.D. from the University of Newcastle upon Tyne. Dr. Quinn completed a postdoctoral fellowship at the University of California San Francisco and is a fellow of the Royal College of Physicians London.

Don Nicholson was most recently chief executive officer of Nimbus. He joined from Merck where he held various strategic, leadership and operational roles in diverse therapeutic areas, including respiratory, inflammation, immunology, bone, endocrine, urology, infectious diseases and neurosciences. He began his career in 1988 at the Merck-Frost Centre for Therapeutic Research in Montreal where he was vice president & site head of the Merck Neurosciences Research site in San Diego, and vice president and worldwide discovery head for the Respiratory & Immunology Franchise, based in Kenilworth, N.J. Don has co-authored more than 150 publications in peer-reviewed scientific and medical journals and is internationally recognized for his contributions to the field of apoptotic cell death. Don received his doctorate in biochemistry from the University of Western Ontario, and trained as a Medical Research Council postdoctoral fellow at the University of Munich in Germany. He is the recipient of multiple academic and professional honors.

Geoff McDonough is chief executive officer of Generation Bio. Geoff formerly served as president and chief executive officer of Swedish Orphan Biovitrum AB (Sobi) from 2011–2017. Prior to Sobi, he held a variety of senior roles at Genzyme Corporation, including president of Genzyme Europe and senior vice president and general manager of the global lysosomal storage disease business. Geoff has a Bachelor of Science in biology and a Bachelor of Arts in philosophy from University of North Carolina at Chapel Hill, graduating summa cum laude in both. He obtained his doctor of medicine at Harvard Medical School and completed his residency training in internal medicine and pediatrics at Massachusetts General Hospital and Boston Children's Hospital.

Jeff Jonas joined Sage as CEO in 2013 and has more than 20 years of experience on both the scientific and business sides of the pharmaceutical and healthcare industries, particularly in the CNS field. Before joining the Sage team, Jeff served as president of the regenerative medicine division of Shire plc and previously as senior vice president of research and development, pharmaceuticals at Shire. Prior to Shire, he served as the executive vice president of ISIS Pharmaceuticals (now known as Ionis Pharmaceuticals), as the chief medical officer and executive vice president of Forest Laboratories, Inc. and in senior-level positions at Upjohn Laboratories. Jeff founded AVAX Technologies, where he served as CEO and president, and SCEPTOR Industries, where he served as chairman, president and chief technology officer. Earlier in his career, he served as independent director at Cara Therapeutics, Inc. and director of AVAX Technologies. He has published more than 70 scientific papers and chapters, authored more than 100 books, scientific articles and abstracts, and has received numerous awards. Jeff received his B.A. from Amherst College and M.D. from Harvard Medical School. He completed a residency in psychiatry at Harvard and then served as chief resident in psychopharmacology at McLean Hospital, Harvard Medical School.

Gustav Christensen is chairman of Morphic Therapeutic, a public biotech company. He was president and chief executive officer of Dyax Corp (NASDAQ: DYAX) from January 1, 2009 until it was acquired by Shire for $6.4 billion in January 2016. He joined Dyax in early 2007 as executive vice president and chief business officer. Gustav began his career at Baxter International, with his last position being vice president of operations of the Fenwal Laboratories division. He left in 1983 to become the vice president of business development at the Genetics Institute from 1983 to 1988. Mr. Christensen received his Master of Science in economics from the University of Aarhus, Denmark and his Master of Business Administration from Harvard Business School.

Jason Rhodes is a partner at Atlas Venture. Jason is the chairman and previously founding chief executive officer of Generation Bio, and a founder and the executive chairman and acting chief executive officer of Disarm Therapeutics. He is on the boards of Replimune, Gemini Therapeutics, Accent Therapeutics, and Bicycle Therapeutics. Prior to joining Atlas Venture in 2014, Jason was president of Epizyme (NASDAQ: EPZM), where he charted the company’s strategic course from a private, research-stage business into the clinic and through a successful IPO in 2013. Prior to Epizyme, Jason led business development at Alnylam (NASDAQ: ALNY). Before Alnylam, Jason was a founder and partner with Fidelity Biosciences (now F-Prime Capital), the life sciences venture capital arm of Fidelity Investments. Jason received his Bachelor of Arts degree from Yale University and his Master of Business Administration from the Wharton School of the University of Pennsylvania. He is a member of the advisory board of the Harvard Institute for RNA Medicine, the BIDMC Board of Overseers, the Blavatnik Fund at Yale University, and the Berklee College of Music Presidential Advisory Council.

Antoinette Paone was previously vice president and head of North American regulatory affairs strategy at Vertex Pharmaceuticals, where she provided regulatory leadership on the marketing application submissions and approvals for Incivek®, Kalydeco®, Orkambi® and Symdeko®. She previously led the Global Regulatory Affairs CMC department at Vertex. Before that, she held multiple positions in chemical research and development and regulatory affairs at Millennium Pharmaceuticals, and began her career as a research chemist at Merck & Co., Inc. Antoinette earned a Bachelor of Science degree in chemistry from Fordham University, a Master of Science in organic chemistry at Yale University and a Master of Business Administration from Bentley College.

Sara has nearly 20 years experience managing human resource and patient advocacy functions in the biotech industry, both from within companies and as an independent consultant and coach. She has a life-long passion for unleashing the best in people and catalysing their collective excellence within organizations. While working at companies such as Genzyme Corporation, Sara drove leadership and organization development strategy from design to implementation. She has a Bachelors of Arts degree in psychology from Bucknell University. She completed the Greater Boston Executive Program at MIT’s Sloan School of Management and holds a professional coaching certification at New Ventures West.

Phillip served as Principal at Atlas Venture from 2016-2018, where he focused on novel platform company creation and development. Phillip played a founding role in the creation of Generation Bio and Dyne Therapeutics, and was a board observer for HotSpot Therapeutics. Phillip was previously director of MRL Ventures Fund at Merck, where he worked to construct the strategic fund and led investments in early-stage therapeutics technologies including Alector, Translate Bio, Spero Therapeutics and miRagen. Prior to that, Phillip was an associate at Flagship Ventures, where he was involved in building several of Flagship’s startups including Codiak BioSciences and Indigo Ag. Phillip graduated from MIT with dual Bachelor of Science degrees in Biological Engineering and Physics, and earned his doctorate in Systems Biology from UCSD, where he was a NSF graduate research fellow.

Jennifer served as the director, intellectual property at the Broad Institute from 2016–2018. Previously, she was the vice president of intellectual property at KindredBio, and spent nearly a decade at Genentech in the IP group, where she was associate general counsel and director of oncology. Jennifer was an associate at Finnegan, Henderson, Farabow, Garrett & Dunner, LLP and a technical specialist at Lahive & Cockfield, LLP; at both firms, her practice focused on preparation and prosecution of patent applications and counseling for life sciences and biotechnology companies. Jennifer received her Juris Doctor degree from Stanford Law School, and her doctorate in microbiology and molecular genetics from Harvard University. She also received a Bachelor of Arts degree in biology from Williams College.

Matt served as vice president, head of chemistry and platform immunology at Moderna from 2014 – 2017. Matt was director and head of RNA medicinal chemistry at Merck, spending 16 years in several roles including small molecule program leadership and head of chemistry capabilities enhancement. He was involved in numerous therapeutic areas including oncology, cardiovascular, neuroscience and infectious disease that spanned a range of modalities including small molecules, siRNA and peptide conjugates. Matt graduated from Virginia Tech with a Bachelor of Science in chemistry and earned his doctorate in chemistry from the University of North Carolina at Chapel Hill, with a focus on physical organic chemistry and natural product synthesis.

Doug served as global development team lead and vice president for neurology at Shire from 2015–2017 where he was responsible for the development of the rare neuroscience programs including the lysosomal storage disorders, neurodegeneration, and gene therapy candidates. Doug served as senior director of corporate strategy and portfolio management at Biogen, where he led the development effort for Alzheimer's disease, amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). Doug led the development of Spinraza™, now approved for SMA. Doug was on the faculty of The Johns Hopkins School of Medicine as associate professor of neurology with joint appointments in the Department of Molecular Microbiology and Immunology and Cellular and Molecular Medicine, and ran a lab that investigated fundamental aspects of motor neuron/axon biology, provided direct patient care and ran clinical trials, mainly spinal cord and neuromuscular disorders. Doug has participated on the boards and scientific advisory boards of several non-profit organizations, including the Transverse Myelitis Association, CureSMA and the ALS Association. Doug received his medical degree from Jefferson Medical College, as well as his doctorate in biochemistry and molecular biology. Doug obtained his Master of Business Administration, with a specialization in entrepreneurship and finance, as well as his Bachelor of Arts degree in biochemistry from Princeton University.

Will was previously chief business officer at Ignyta, a company focused on rare, genetically defined cancers. He held this role from 2015 until the acquisition of Ignyta by Roche in 2018. Before that, he was vice president of corporate development at Foundation Medicine and held business development and commercial roles at Halozyme Therapeutics, Biogen Idec and Neurocrine Biosciences. He began his career as a strategy consultant at firms including IMS Health and Deloitte. Will obtained a Master of Business Administration from London Business School and a Bachelor of Arts degree in economics from the University of Exeter.

Mark was senior vice president of pharmaceutical sciences and rare disease franchise head at bluebird bio Inc. from 2012 until 2016. Previously, he was senior director of research and development and site head in Cambridge, Massachusetts for Baxter Healthcare Corporation from 2010 to 2012. Prior to 2010, Mark worked at Archemix, Momenta Pharmaceuticals, Millennium Pharmaceuticals, and DuPont Pharmaceuticals. His experience spans from development through commercialization including the first commercial launch for a bioequivalent (generic Lovenox™), the ANDA submission for generic Copaxone™, the sNDA for Integrilin™, and the BLA for Zynteglo™ as well as numerous INDs across small-molecules, biologics, peptides, biologics, complex mixture, and gene therapy products. Mark holds a doctorate in chemical engineering and a Master of Science in chemical engineering practice from the Massachusetts Institute of Technology, and obtained his Bachelor of Science in chemical engineering from The Cooper Union.

Matt was previously chief financial officer at SomaLogic, Inc., a Colorado-based biotech. In that role, he helped lead a complete strategic overhaul for the company, closed several large business development deals and oversaw strong commercial growth both in the U.S. and overseas. Prior, Matt served as senior equity analyst at Marsico Capital Management, where he was the firm’s primary health care investment specialist. Matt began his career as a practicing anesthesiologist. He earned a Master of Business Administration from Columbia Business School, a medical degree from University of Maryland School of Medicine, and a Bachelor of Arts degree from St. Mary’s College of Maryland.

Geoff formerly served as president and chief executive officer of Swedish Orphan Biovitrum AB (Sobi) from 2011– 2017. Prior to Sobi, he held a variety of senior roles at Genzyme Corporation, including president of Genzyme Europe and senior vice president and general manager of the global lysosomal storage disease business. Geoff has a Bachelor of Science in biology and a Bachelor of Arts in philosophy from University of North Carolina at Chapel Hill, graduating summa cum laude in both. He obtained his doctor of medicine at Harvard Medical School and completed his residency training in internal medicine and pediatrics at Massachusetts General Hospital and Boston Children's Hospital.