Katherine Ann High

Focuses on development of gene therapies for genetic disease, most notably the effective translation of recombinant AAV vectors. Using hemophilia as a model, achieved long-term cures in animal models. Translated experimental approach safely into hemophilia patients and defined and helped to solve problems that posed obstacles to long-term cures in humans. Extended these approaches to children and adults with a rare form of congenital blindness. Recipient, National Hemophilia Foundation Researcher of the Year Award, Foundation Fighting Blindness Board of Directors' Award, Outstanding Achievement Award of the American Society of Gene and Cell Therapy. Co-founder and President of Spark Therapeutics. Achieved first FDA approval of a gene therapy for genetic disease in 2017, for a rare form of congenital blindness. Led teams that received FDA Breakthrough Therapy designation on 3 investigational gene therapies.  Led team that received 2019 Prix Galien Award USA for Best New Biotechnology Drug.