Maria Grazia Roncarolo

A pediatric immunologist by training, Dr. Maria Grazia Roncarolo is a pioneer in cell and gene therapy for genetic diseases, and a world renowned expert in immune tolerance. Her breakthrough translational research intersects two distinct, but interrelated areas: inducing immune tolerance following hematopoietic stem cell transplantation (HSCT) and designing novel gene therapies for patients with genetic diseases of the hematopoietic and immune systems.

Dr. Roncarolo discovered the immunoregulatory and suppressive Type 1 regulatory T (Tr1) cells and brought them to the clinic to prevent Graft-versus-Host Disease (GvHD), a major complication of otherwise life-saving allogeneic HSCT. In addition, she successfully led the first stem cell-based gene therapy trial for severe combined immunodeficiency (SCID) patients lacking adenosine deaminase (ADA-SCID; “bubble boy disease”). The trial, combining gene corrected blood stem cells with low-dose chemotherapy, obtained Orphan drug status from the FDA and EMEA, and in May 2016 received European Commission approval to market as “Strimvelis”. This major milestone enabled her to spearhead a team designing and implementing stem cell and gene therapies for other severe inherited diseases, including Wiskott-Aldrich Syndrome (WAS) and Metachromatic Leukodystrophy (MLD).

Dr. Roncarolo is currently the George D. Smith Professor of Pediatrics (Stem Cell Transplantation) and Medicine (Blood and Marrow Transplantation), Co-Director of the Institute for Stem Cell Biology and Regenerative Medicine (ISCBRM), and founder and Director of the Center for Definitive and Curative Medicine (CDCM) at Stanford School of Medicine where she leads efforts to translate scientific discoveries in hematologic malignancies and regenerative medicine into patient therapies.