Maria Grazia Roncarolo

Dr. Maria Grazia Roncarolo is one of the world’s foremost experts in gene therapy and one of Graphite Bio’s academic founders. She is recognized globally for her leadership in translating scientific discoveries in genetic diseases and regenerative medicine into novel patient therapies, including the world’s first ex vivo gene therapy. The George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine at Stanford University, Dr. Roncarolo established the Stanford Center for Definitive and Curative Medicine to cure patients with currently incurable diseases through the development of innovative stem cell and gene-based therapies. During her earlier tenure as director of the Telethon Institute for Gene Therapy at the San Raffaele Scientific Institute in Milan, Dr. Roncarolo developed novel approaches in gene therapy. Her work at the institute led to the discovery of ex vivo gene therapies for genetic diseases of the immune system, including ADA-SCID and WASP, and metabolic diseases such as metachromatic leukodystrophy. The landmark stem cell gene therapy treatment for ADA-SCID was the world’s first to be approved by the European Medicines Agency (EMA), under the brand name Strimvelis®, in May 2016.