Rosa Bacchetta

Rosa Bacchetta’s professional goal as a pediatrician specializing in immunology, has been to challenge the limits of "inexplicable" and "untreatable" diseases. Her translational research focuses on Autoimmune Genetic Diseases and Primary Immunodeficiencies, from understanding the pathogenesis, defining novel diagnostic and prognostic markers and investigating new therapeutic approaches. Following the completion of her Medical School and Residency in Pediatrics at the University of Turin, she received training in molecular and cellular immunology in the United States (DNAX Research Institute of Molecular and Cellular Biology, Palo Alto). There, she was first exposed to the importance of integrating in depth laboratory research with clinical observations to develop a translational research approach to science. She then worked for fifteen years at the San Raffaele Scientific Institute (HSR-TIGET), where she focused on dissecting the genetic and immunological basis of primary immune-regulatory diseases that might be treated by gene therapy. She pioneered the study of Immune Dysregulation-Polyendocrinopathy-Enteropathy-X linked (IPEX) Syndrome, the prototype genetic disease of thymic-derived T regulatory (Treg) lymphocytes deficiency. She is Faculty at Stanford University School of Medicine since 2015. At Stanford, she is currently conducting late stage pre-clinical IND enabling studies for an innovative Treg cell therapy-based treatment for IPEX patients and, at the same time, she is investigating how to correct FOXP3 gene mutations in IPEX patients’ hematopoietic stem cells. Improving therapies for autoimmune genetic diseases will lead to treatment of more common immune-mediated diseases.