Session Abstract – PMWC 2022 Silicon Valley

 Session Chair Profile

Ph.D., Assistant Professor, UCSF

Biography
Dr. Paulk’s lab is focused on next-generation AAV gene delivery and gene editing platforms. They are species/disease/organ/tissue/pathway/cell type/gene agnostic, working at the platform level to forge new paths engineering novel ways to make all AAV therapies cheaper, easier and faster. Dr. Paulk has developed gene therapies for numerous rare diseases and cancer, and has applied high-throughput comparative proteomic and epigenomic approaches to uncover fundamental AAV biology. Dr. Paulk sits on the Scientific Advisory Boards for Dyno Tx, CEVEC, Excision Bio, WhiteLab Genomics and Sarepta; Scientific Editorial Boards for the journals Human Gene Therapy and Gene Therapy; and currently sits on the American Society of Gene & Cell Therapy Translational Science & Product Development Committee. Paulk earned her BS in Medical Microbiology, her PhD in Viral Gene Therapy from Oregon Health & Science University, and completed her Postdoctoral Fellowship in Human Gene Therapy at Stanford University.


 Speaker Profile

M.D., Ph.D., Professor of Pediatrics, Stanford University School of Medicine

Biography
Matthew Porteus is the Sutardja Clark Professor of Definitive and Curative Medicine in the Department of Pediatrics and Institute of Stem Cell Biology and Regenerative Medicine at Stanford. He is the co-Director for the Stanford Center for Definitive and Curative Medicine (CDCM). His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood and immune system (including sickle cell disease) and other organ systems. His goal is to combine his research and clinical interests to develop innovative curative therapies. His dream is to one day develop gene editing so that patients are cured of their disease before they or their parents even knew they had it. He served on the 2017 National Academy Study Committee of Human Genome Editing and currently serves on the Scientific Advisory Board for WADA on Cell and Gene Doping and the NIH NExTRAC advisory committee evaluating the emergence of new technologies.

Talk
Genome Editing of Stem Cells
Genome editing provides unprecedented precision in developing personalized medicines for patients. By engineering cells ex vivo with high precision, it may be possible to generate cures for both rare and common diseases. I will discuss our progress towards this ambitious goal.


 Speaker Profile

M.D., Chief Medical Officer, Ultragenyx Gene Therapy and Inborn Errors of Metabolism

Biography
Dr. Crombez joined Ultragenyx following the acquisition of Dimension Therapeutics, a liver directed AAV gene therapy company. During his tenure at both companies, Dr. Crombez led the clinical development efforts for four clinical stage programs including gene therapy programs for hemophilia B, hemophilia A (in partnership with Bayer), ornithine transcarbamylase (OTC) deficiency and glycogen storage disorder type Ia (GSDIa). Dr. Crombez is also serving as an Industry Representative on the FDA Cellular, Tissue, and Gene Therapies Advisory Committee. Prior to this work in gene therapy, Dr. Crombez worked at Shire in their Human Genetics Therapy business unit for nearly eight years. Prior to joining industry, Dr. Crombez was Assistant Professor, Department of Pediatrics, Division of Medical Genetics at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA).


 Speaker Profile

M.D., Ph.D., Chief Scientific Officer, 4D Molecular Therapeutics

Biography
Peter Francis, M.D., Ph.D., served as our Chief Medical Officer January 2019 through February 2020 before becoming our Chief Scientific Officer. Dr. Francis previously served as our Senior Vice President, Clinical Translational R&D, and Retina Therapeutic Area Head from August 2018 to January 2019. Dr. Francis previously served as Chief Medical Officer at RetroSense Therapeutics from February 2012 until August 2016 when it was purchased by Allergan Inc. Dr. Francis practices as a physician at Orion Eye Center. Dr. Francis received his B.Sc. in Molecular Cell Biology from the University of Southampton, England in 1991. He earned his M.D. from the University of Southampton, England in 1992. He earned his Ph.D. in ophthalmic genetics from University College, London in 2000.


 Speaker Profile

Ph.D., Co-Founder and Vice President, Forge Biologics

Biography
Dr. De Silva is an experienced scientist and entrepreneur, with more than a decade of experience in discovery research, therapeutic development, and partnering across oncology, infectious disease, and gene therapy. Prior to founding Forge, she held R&D and leadership roles in both academic and industry settings, including at Genentech, Ohio State University and Myonexus Therapeutics.


 Speaker Profile

Ph.D., Chief Scientific Officer, Q2 Solutions

Biography
Dr. Patrice Hugo is responsible for the medical affairs and scientific activities for the Central Laboratories, and Specialty Testing Centers of Excellence worldwide including the Expression Analysis Genomics, Vaccine, Biomarkers Translational Science and Innovation Laboratory and BioAnalytical/ADME laboratories. Prior to joining Q² Solutions, Dr. Hugo was Associate Vice President and Chief Clinical Trial Scientist at LabCorp/Covance Central Lab Division and Chief Scientific Officer at Clearstone Central Lab acquired by LabCorp. Previously, he worked at Caprion (now CellCarta) as Executive Vice President R&D, and Principal Investigator at the Montreal Clinical Research Institute with academic affiliation at McGill University and University of Montreal. Dr. Hugo obtained his Ph.D. in Experimental Medicine & Immunology at McGill University and held post-doctoral positions at the Walter and Eliza Hall Institute, Australia, and the Howard Hughes Medical Institute, USA. He is author or co-author of over 75 scientific manuscripts and has over 25 years of biomarker experience.