Alessandra Biffi, MD is is a pediatric hematologist and stem cell transplant physician, specialized in studying gene therapy approaches to treat childhood genetic diseases. She is a professor of pediatrics at the University of Padua and the chief of the Pediatric Onco-hematology Unit at Padua Hospital.

Her studies focused on lysosomal storage genetic diseases. In particular, her research on metachromatic leukodystrophy, a very severe neurodegenerative disease, progressed to advanced clinical development. Since 2015, she has been the director of the gene therapy program at Dana-Farber/Boston Children’s Cancer Center in Boston, USA. She returned to Italy in 2018, where she is actively involved in gene therapy studies for neurological genetic diseases, hemoglobinopathies, immunodeficiencies and cancer.