Dr. Katherine A. High is a physician-scientist whose pioneering research has helped shape the field of modern gene therapy. She is Professor Emerita of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania, Co-Founder of Spark Therapeutics, and currently serves as CEO of RhyGaze, Inc.

Dr. High was formerly an Investigator of the Howard Hughes Medical Institute at The Children’s Hospital of Philadelphia (CHOP), where her groundbreaking research on gene therapy for hemophilia led to pivotal discoveries about the human immune response to adeno-associated viral (AAV) vectors across various tissues. Her work was instrumental in translating gene therapy from the lab to the clinic for multiple inherited disorders.

At Spark Therapeutics, Dr. High led the development of Luxturna®, the first FDA-approved gene therapy for a genetic disease, which restored vision in patients with inherited retinal blindness. For this historic achievement, she was co-recipient of the Sanford Lorraine Cross Award for Medical Innovation in 2018. She also led the early clinical trials of Beqvez®, now approved for hemophilia B, and obtained FDA Breakthrough Therapy designation for Spark’s clinical program in hemophilia A—further cementing her legacy as a leader in genetic medicine.

Dr. High has held prominent leadership roles, including serving as President of the American Society of Gene and Cell Therapy and as a member of the FDA Advisory Committee on Cell, Tissue, and Gene Therapy. Her scientific contributions have been recognized through election to the National Academy of Medicine and the National Academy of Sciences. A granddaughter of two Philadelphians, she is particularly honored to be named a Distinguished Daughter of Pennsylvania.