Katherine High is recognized for her pioneering research and development of gene therapies for genetic disease. She is known for her studies in adeno-associated viral (AAV)-mediated gene therapy for hemophilia, including the development of AAV vectors that brought about long-term improvement in small and large animal models of hemophilia. She translated these findings to studies of men with severe hemophilia, and overcame hurdles identified in clinical trials including the risk of germline transmission and human immune response to the AAV vector.
Dr. High earned her undergraduate degree in chemistry at Harvard, her M.D. at the University of North Carolina (UNC) and completed medical training in hematology at Yale. After serving on faculties at UNC and at the University of Pennsylvania School of Medicine and the Children’s Hospital of Philadelphia, where she was an Investigator of the Howard Hughes Medical Institute, she left the University to co-found Spark Therapeutics, where she led the team that earned the first FDA approval of a gene therapy for a genetic disease, a rare form of inherited blindness. She is currently President of Therapeutics at Asklepios BioPharmaceuticals. She is a past president of the American Society of Gene and Cell Therapy, and a member of both the National Academy of Medicine and the National Academy of Sciences.

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Primary Section

Section 41: Medical Genetics, Hematology, and Oncology

Secondary Section

Section 43: Immunology and Inflammation