George D. Smith Professor in Stem Cell and Regenerative Medicine, Emerita · Co-Founder, President & Head of R&D, Tr1X, Inc. · Stanford University / Tr1X, Inc. · San Diego
Maria Grazia Roncarolo, MD, President and Head of R&D Tr1X Inc., George D Smith Professor of Stem Cell transplantation and Regenerative medicine, Emerita, Stanford University.
Maria Grazia Roncarolo is a pediatric immunologist by training and an expert in cell and gene therapy. She is recognized globally for her leadership in translating scientific discoveries in immune-mediated diseases and regenerative medicine into novel patient therapies, including the world’s first ex vivo gene therapy. During her tenure at Stanford University as the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine, Dr. Roncarolo established the Stanford Center for Definitive and Curative Medicine to cure patients with currently incurable diseases through the development of innovative 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 a pipeline of gene therapy products to cure rare genetic diseases, including Strimvelis® and Lenmeldy®. She discovered a new class of immunoregulatory and suppressive T cells, called type 1 regulatory T (Tr1) cells, and led phase I clinical trials using these cell therapeutics to prevent graft-versus-host disease (GvHD), a major complication of otherwise life-saving allogeneic HSCT. She is the scientific founder of Tr1X Inc., which is developing a new class of engineered Tr1 cell-based therapies for inflammatory and autoimmune diseases.