Katherine High, MD

Katherine A. High, MD

Professor Emerita, Perelman School of Medicine
University of Pennsylvania

Dr. Katherine A. High is a physician-scientist with a longstanding interest in gene therapy for genetic disease. As an investigator of the Howard Hughes Medical Institute at the Children's Hospital of Philadelphia (CHOP) and a faculty member of the University of Pennsylvania School of Medicine, she carried out pioneering basic and clinical studies in gene therapy for hemophilia and for a rare form of retinal dystrophy caused by mutations in the RPE65 gene. As co-founder, president and head of R&D at Spark Therapeutics, she led the approval of the first gene therapy for a genetic disease in the United States. Spark has received FDA breakthrough therapy designations for three different therapeutics, including for retinal dystrophy due to RPE65 mutations and for hemophilia B and hemophilia A.  Dr. High is an elected member of the National Academy of Medicine, the American Academy of Arts and Sciences, and is a fellow of the Faculty of Pharmaceutical Medicine of the Royal College of Physicians (London). Dr. High also serves on the board of directors of CRISPR Therapeutics.