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Maria Grazia Roncarolo MD
Professor of Pediatrics and Medicine
Stanford University School of Medicine
USA
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Dr. Maria Grazia Roncarolo is one of the world’s foremost experts in translational medicine and a pioneer in cell and gene therapy. She is recognized globally for her leadership in translating scientific discoveries in genetic diseases and regenerative medicine into novel patient therapies. 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.
A pediatric immunologist by training, she earned her medical degree at the University of Turin, Italy. Dr. Roncarolo worked at DNAX Research Institute for Molecular and Cellular Biology in Palo Alto, where she contributed to the discovery of novel cytokines, cell-signaling molecules that are part of the immune response.
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. She is a co-founder of Graphite Bio, which is developing a new class of therapies to correct by gene editing genetic defects in people with serious and life-threatening diseases of the blood.
She discovered a new class of T cells, called T regulatory type 1 (Tr1) cells, and is leading clinical trials using these cell therapeutics to prevent immune mediated diseases. She recently started a new biotech company (Tr1X Bio), which is developing a new class of Tr1 cell-based therapies for autoimmune and inflammatory diseases.
A pediatric immunologist by training, she earned her medical degree at the University of Turin, Italy. Dr. Roncarolo worked at DNAX Research Institute for Molecular and Cellular Biology in Palo Alto, where she contributed to the discovery of novel cytokines, cell-signaling molecules that are part of the immune response.
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. She is a co-founder of Graphite Bio, which is developing a new class of therapies to correct by gene editing genetic defects in people with serious and life-threatening diseases of the blood.
She discovered a new class of T cells, called T regulatory type 1 (Tr1) cells, and is leading clinical trials using these cell therapeutics to prevent immune mediated diseases. She recently started a new biotech company (Tr1X Bio), which is developing a new class of Tr1 cell-based therapies for autoimmune and inflammatory diseases.