Katherine A. High, MD

Dr. Katherine High is a leader in gene therapy for genetic disease, with a career spanning academic medicine, clinical research, and biotechnology innovation. She spent much of her career as a faculty member of the Perelman School of Medicine at the University of Pennsylvania and simultaneously served as an Investigator of the Howard Hughes Medical Institute at the Children’s Hospital of Philadelphia. There, she led groundbreaking bench-to-bedside studies in gene therapy for hemophilia, including the first clinical trials using AAV vectors delivered to human skeletal muscle and liver. 

Her work has been instrumental in advancing understanding of the human immune response to AAV gene delivery, and she developed strategies now widely adopted across the field to improve safety and efficacy. In addition to her research contributions, Dr. High served a five-year term on the U.S. FDA Advisory Committee on Cell, Tissue, and Gene Therapies and is a past president of the American Society of Gene & Cell Therapy.

In 2013, Dr. High co-founded Spark Therapeutics, where she served as President, Chief Scientific Officer, and a member of the Board of Directors. Under her leadership, Spark achieved the first FDA approval of a gene therapy for an inherited disease: Luxturna, a treatment for a rare form of genetic blindness. She also led development programs for gene therapy in hemophilia B, culminating in an approved therapy (Beqvez) following a Pfizer-sponsored Phase 3 trial. In December 2019, she played a key role in Spark’s $4.8 billion acquisition by Roche. In February 2020, she left Spark Therapeutics and subsequently joined the boards of CRISPR Therapeutics and Incyte BioPharmaceuticals.

From January 2021 to December 2022, Dr. High served as President of Therapeutics at AskBio, a wholly owned subsidiary of Bayer AG focused on gene therapy. In January 2023, Dr. High became a Visiting Professor at Rockefeller University and served as a Life Sciences Advisor to GV (Google Ventures). In December 2024, she assumed the role of CEO of RhyGaze AG, a venture-backed, early-stage gene therapy company focused on gene therapy for retinal disease.

Dr. High earned her bachelor’s degree in chemistry from Harvard University, her MD from the University of North Carolina School of Medicine, and completed business training through the University of North Carolina’s Management Institute for Hospital Administrators. She is an elected member of the National Academy of Medicine, 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).