Katherine A. High MD

Spark Therapeutics

Dr. Katherine High is board certified in internal medicine and hematology and was a Professor at the Perelman School of Medicine at the University of Pennsylvania for 22 years. In 2002 she was appointed an Investigator of the Howard Hughes Medical Institute, and in 2004 became the Founding Director of the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia. Dr. High has a long-standing interest in gene therapy for genetic disease and has pioneered their safe and effective translation to clinical studies. In 2014, she co-founded a gene therapy company, Spark Therapeutics, where she currently serves as President and Head of R&D. Under Dr. High’s leadership, Spark Therapeutics received FDA approval of voretigene neparvovec, the first AAV gene therapeutic to be licensed in the US, and the first pharmacologic treatment for an inherited retinal dystrophy. Spark has achieved FDA Breakthrough Therapy Designation for three programs, including two in hemophilia, in addition to the program that led to the licensing of voretigene. Dr. High is an elected member of the National Academy of Medicine and the American Academy of Arts and Sciences. She has published over 200 scientific papers and holds a number of patents related to gene therapy. Dr. High is the past recipient of the National Hemophilia Foundation Researcher of the Year Award (2000), and was elected as an AAAS Fellow for “Distinguished contributions in the field of human gene therapy” in 2000. She received the 2003 Distinguished Alumna Award from the University of North Carolina at Chapel Hill, and the 2010 Outstanding Achievement Award from the American Society of Gene and Cell Therapy. She received the Foundation for Fighting Blindness Board of Directors Award (2007) and the E. Donnall Thomas Award of the American Society of Hematology (2013). Dr. High is an active member of the American Society of Hematology, and of the American Society of Gene and Cell Therapy, of which she is a Past President (2004-05). Dr. High previously served on the National Heart, Lung, and Blood Institute (NHLBI) Advisory Council, the NHLBI Gene Therapy Resource Program (GTRP) Steering Committee, and the FDA Cellular, Tissue and Gene Therapies Advisory Committee (CTGAC). She currently serves as a member of the Board of Scientific Counselors of the NIH National Eye Institute.