Dr. Katherine High, an internist and hematologist by training, began her career studying the molecular basis of blood coagulation and the development of novel therapeutics for the treatment of bleeding disorders. Her pioneering bench-to-bedside studies of gene therapy for hemophilia led to a series of studies that characterized the human immune response to gene delivery vectors. Her work evolved to encompass clinical translation of genetic therapies for multiple inherited disorders, and as the Founding Director of the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia, Dr. High assembled a multidisciplinary team of scientists and researchers working to discover new gene and cell therapies for genetic diseases and to facilitate rapid translation of preclinical discoveries into clinical application. At CHOP and Penn, Dr. High was an Investigator of the Howard Hughes Medical Institute, and held an endowed chair at the medical school.
In 2013, Spark Therapeutics, a biotechnology company based in Philadelphia, was formed based on programs that Dr. High had led at Children’s Hospital and in 2014, she joined Spark full-time as President and Head of R&D; in 2019, Spark was acquired by Roche. While at Spark, she led the team that achieved the first FDA approval of a gene therapy for a genetic disease in the US, for a rare form of congenital blindness, and led the development of a gene therapy for hemophilia B that has now completed Phase 3 testing. Dr. High served a five-year term on the FDA Advisory Committee on Cell, Tissue and Gene Therapies and is a past-president of the American Society of Gene & Cell Therapy (ASGCT).
She received her A.B. in chemistry from Harvard University, an M.D. from the University of North Carolina School of Medicine, and business certification from the UNC Business School Management Institute for Hospital Administrators. She currently serves on the Board of Directors of CRISPR Therapeutics (NASDAQ: CRSP), and Incyte Corporation (NASDAQ:INCY), and on the Board of Trustees of the College of Physicians of Philadelphia. She is the recipient of many honors and awards, is the author of more than 200 scientific papers, and the holder of multiple issued patents on gene therapy. She is an elected member of the National Academy of Medicine (US), the American Academy of Arts and Sciences, the Faculty of Pharmaceutical Medicine of the Royal College of Physicians (London), and the National Academy of Sciences (US). She is currently a Visiting Professor at Rockefeller University in New York.