Dean of Harvard Medical School. Professor of Medicine and BIological Chemistry at HMS and Boston Children's Hospital. Research focuses on stem cells and cancer pathways in hematologic malignancies and blood development.
Adventures in ex vivo and in vivo muscle stem cell gene editing
Dr. Pyle is a Professor and Vice Chair in the Department of Microbiology, Immunology and Molecular Genetics, and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research (BSCRC) at UCLA. Dr. Pyle's lab uses multi-disciplinary approaches to study muscle stem cells, human pluripotent stem cells and differentiation. Dr. Pyle's lab studies both basic aspects of stem cell biology, reprogramming, and muscle development as well as more translational aspects to develop therapeutic approaches for patients with Duchenne Muscular Dystrophy (DMD). The lab is interested in developing ex vivo and direct in vivo CRISPR correction stem cell strategies for DMD. Dr. Pyle is also co-founder of MyoGene Bio which aims to develop a therapeutic CRISPR/Cas9 platform platform designed to restore the reading frame of the DMD gene.
William Pu, MD, is the Director of Basic and Translational Cardiovascular Research in the Department of Cardiology at Boston Children’s Hospital, and the Aldo R. Castañeda Professor of Pediatrics at Harvard Medical School. Dr. Pu has broad expertise in cardiac biology that includes cardiac development, heart failure, cardiac regeneration and in vitro cardiac disease modeling. His lab has made fundamental discoveries in gene regulation in developing and diseased hearts, particularly in the area of transcriptional regulation. His lab is currently studying the pathogenesis of inherited heart diseases and using insights gained to developed targeted therapies.
Dr. Pu completed his combined BS-MS degree at Yale University and obtained his MD degree from the Harvard Medical School/MIT Science and Technology Program in 1993. He trained in Pediatrics and Pediatric Cardiology at Boston Children's Hospital. He received his training in basic research in the laboratories of Kevin Struhl, David Clapham, and Seigo Izumo. He established an independent research lab at Boston Children’s in 2004.
Executive Vice President, Head of Research and Development, CRISPR Therapeutics
Dr. Tony Ho is Head of Research and Development at CRISPR Therapeutics over seeing development of allogenic CAR-T, hemoglobinopathy and regeneration medicine. 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 and Imfinizi (anti-PD-L1), AstraZeneca’s first immuno-oncology drug. 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 and is co-inventor of 14 patents. He is currently adjunct Associate Professor of Neurology at University of Pennsylvania and Assistant Professor (Part-time) of Neurology at Johns Hopkins University.