Co-founder, President and Chief Scientific Officer, Spark Therapeutics, Inc.
Dr. Katherine A. High is a hematologist with a longstanding interest in the development of gene therapy for the treatment of genetic disease. After a residency in internal medicine, she trained with Dr. Ed Benz as a fellow in hematology at Yale. She pursued a career in basic and translational research as a faculty member at UNC-Chapel Hill and at the University of Pennsylvania. Dr. High’s initial research interests focused on delineating the molecular defects responsible for hemophilia. Subsequently she sought to establish proof-of-concept in the canine model for a gene therapy approach to hemophilia, but was stymied by the inability to produce enough recombinant vector to treat a 20 kg dog.
After a lunchtime conversation with Y.W. Kan when he was a visiting professor at Penn, she established a collaboration with a gene therapy company in San Francisco that led to successful cure of hemophilia B in the dog model. Since then, her research has focused on safe and effective clinical translation of genetic therapies for inherited disorders. In 2002 she was named an HHMI Investigator, and in 2004, was named the Founding Director of a Center at The Children’s Hospital of Philadelphia focused on gene-based therapies for genetic disease. Trials led by Dr. High at the Center formed the nidus of Spark Therapeutics, a gene therapy company that was spun out of CHOP in March 2013. In September 2014, Dr. High assumed the role of President and Chief Scientific Officer at Spark.
Dr. High has been elected to the Institute of Medicine (IOM) and the American Academy of Arts & Sciences. She is a past president of the American Society of Gene Therapy, and a past recipient of the E. Donnall Thomas Award of the American Society of Hematology. She is delighted to be participating in this symposium honoring Dr. Kan for his seminal contributions in hematology and regenerative medicine.