Encouraging signs for a new generation of AAV-driven gene therapy

Dr Katherine High trained in internal medicine, hematology, and molecular genetics, and began her faculty career at UNC-Chapel Hill. After moving to the University of Pennsylvania, she conducted pioneering bench-to-bedside studies of gene therapy for hemophilia. These led to a series of basic and clinical investigations that characterized the human immune response to AAV gene delivery vectors. Her work evolved to encompass clinical translation of genetic therapies for multiple inherited disorders. As the inaugural director of a Center at The Children’s Hospital of Philadelphia, where she was also an Investigator of the Howard Hughes Medical Institute (HHMI), Dr High assembled a multidisciplinary team of scientists and physicians to discover and develop new gene therapies for genetic diseases. In 2013, her Center at CHOP spun out as Spark Therapeutics, where she led the team that achieved the first FDA approval of a gene therapy for a genetic disease. After Spark was acquired by Roche in 2019, Dr High did a (virtual) sabbatical as a Visiting Professor at Rockefeller University, and in 2021 joined AskBio as President, Therapeutics. Dr High is an elected member of the National Academy of Medicine (US), the American Academy of Arts and Sciences, the Royal College of Physicians (London), and the National Academy of Sciences (US).