James M. Wilson, MD, PhD
Founder and Chairman of Scientific Advisory Council, Dimension
Professor, Medicine, University of Pennsylvania
James M. Wilson, M.D., Ph.D., is the Chair, Director, Gene Therapy Program and Professor of Medicine at the Perelman School of Medicine at the University of Pennsylvania. Dr. Wilson has dedicated his career to gene therapy and much of his research has focused on the development of AAV vectors as vehicles for gene delivery. Prior to joining Penn more than 20 years ago, Dr. Wilson held a faculty position at the University of Michigan, where he started his studies in gene therapy. Earlier, he completed a residency in Internal Medicine at the Harvard-affiliated Massachusetts General Hospital and a postdoctoral fellowship at MIT. Dr. Wilson holds an M.D. and a Ph.D. from the University of Michigan and a B.S. in Chemistry from Albion College.
Ian Alexander, BMedSci, MBBS (Hons), PhD, FRACP (Paeds)
Scientific Advisory Council
Director Laboratory Research, Senior Staff Specialist, The Children’s Hospital at Westmead (CHW)
Professor in Pediatrics and Molecular Medicine, University of Sydney
Ian Alexander, BMedSci, MBBS (Hons), Ph.D., FRACP (Paeds), HGSACG, FAHMS, is Director Laboratory Research and Senior Staff Specialist, The Children’s Hospital at Westmead (CHW), and Professor in Pediatrics and Molecular Medicine, University of Sydney. He is also head of the Gene Therapy Research Unit, a joint initiative of Children’s Medical Research Institute and CHW. During his career, Dr. Alexander headed the first group in Australia to treat a genetic disease (SCID-X1) by gene therapy. After completing specialty training in pediatrics at Prince of Wales Children’s Hospital, Dr. Alexander obtained a Ph.D. in Molecular Biology from the Garvan Institute in Sydney before completing clinical genetics training at the Murdoch Institute in Melbourne. He then undertook postdoctoral studies at the Fred Hutchinson Cancer Research Center in Seattle before returning to Australia to take up his current position. In 2015 he was made a Fellow of the Australian Academy of Health and Medical Sciences.
Terrence R. Flotte, MD
Celia and Isaac Haidak Professor in Medical Education, dean of the School of Medicine and provost and executive deputy chancellor of the University of Massachusetts Medical School (UMMS)
Terence R. Flotte, M.D., serves as the Celia and Isaac Haidak Distinguished Professor, Dean, Provost and Executive Deputy Chancellor, and Chief Research Officer of the University of Massachusetts Medical School (UMMS). An internationally known pioneer in human gene therapy, Dr. Flotte leads a research group that investigates the delivery of genetic diseases that affect children, mainly cystic fibrosis. In 1995, Dr. Flotte and his colleagues became the first to use the adeno-associated virus, or AAV, as a vehicle to deliver corrective genes to targeted sites in the body. He earned his undergraduate degree summa cum laude in the biological sciences from the University of New Orleans and his medical degree from the Louisiana School of Medicine.
Emil D. Kakkis, MD, PhD
President and Chief Executive Officer, Ultragenyx Pharmaceutical
Emil Kakkis, M.D., Ph.D., is Founder, President and Chief Executive Officer of Ultragenyx Pharmaceuticals Inc., a biotechnology company developing therapeutics for rare diseases. Dr. Kakkis also founded the EveryLife Foundation for Rare Diseases (EFRD) to improve regulatory policy issues. The EFRD succeeded in getting legislation into FDASIA that could improve access to the accelerated approval pathway. Prior to founding Ultragenyx and EFRD, Dr. Kakkis joined BioMarin in 1998, where he guided the development and approval of two more treatments for rare disorders, MPS VI and PKU, and contributed to the initiation of seven other treatment programs for rare disorders, three of which are now in clinical development or approved. Dr. Kakkis is board certified in both Pediatrics and Medical Genetics, received his undergraduate degree from Pomona College and has a doctorate degree from the University of California, Los Angeles.
Nicola Longo, MD, PhD
Professor and Chief, Division of Medical Genetics
Director, Fellowship Training Program, Biochemical Genetics,
University of Utah
Nicola Longo, M.D., Ph.D., serves as the Professor and Chief of the Division of Medical Genetics at the University of Utah. He is also Director of the Metabolic Service, Co-Director of the Biochemical Genetics Laboratory, and Director of the Fellowship Training Program in Biochemical Genetics at the University of Utah. He is an expert in inherited metabolic diseases and is intimately involved in the treatment of patients with these diseases at Primary Children’s Hospital. Dr. Longo earned his M.D. and Ph.D. in Molecular Biology at the University of Parma School of Medicine. He received residency and fellowship training in pediatrics, medical genetics, and clinical biochemical genetics at Emory University in Atlanta, Georgia.
Dimension’s Clinical Advisors
Mark Batshaw, MD
Chief Academic Officer, Physician-in-Chief, Children’s National Health System
Director, Children’s Research Institute
Chairman, Pediatrics and Associate Dean, Academic Affairs, George Washington University School of Medicine and Health Sciences
Mark Batshaw, M.D., is Chief Academic Officer and Physician-in-Chief of the Children’s National Health System, where he also serves as Director of the Children’s Research Institute. He is also Chairman of Pediatrics and Associate Dean for Academic Affairs at the George Washington University School of Medicine and Health Sciences. Dr. Batshaw’s research focus is on inborn errors of urea synthesis, in which he is considered an international authority in the development and testing of innovative therapies. He completed his undergraduate degree at the University of Pennsylvania and attended medical school at the University of Chicago. He also completed his residency in pediatrics at the Hospital for Sick Children/University of Toronto in Toronto, Ontario, and his post-doctoral fellowship in neurodevelopmental pediatrics at the Kennedy Institute of Johns Hopkins University School of Medicine.
David A. Weinstein, MD, MMSc
Professor, Director of the Glycogen Storage Disease Program, University of Florida College of Medicine
Knight of the Order of the Smile
Member of the Rare Disease Research Hall of Fame
David A. Weinstein, M.D., M.M.Sc., is Director of the Glycogen Storage Disease Program and Professor of Pediatric Endocrinology at Shands Hospital at the University of Florida. Previously, he was Director of the Glycogen Storage Disease Program at Children’s Hospital Boston. Dr. Weinstein has been inducted in the Rare Disease Research Hall of Fame and was awarded a United Nations recognized international humanitarian award for his efforts to help children with glycogen storage disease from around the world. Following his graduation from Trinity and Harvard Medical School, Dr. Weinstein completed a residency, chief residency, and fellowship in pediatric endocrinology at Children’s Hospital Boston. He subsequently obtained a Masters in clinical investigation from Harvard and MIT.