As a molecular biologist-geneticist by training, Fulvio Mavilio started to focus on hematology and thus blood diseases in the early 1980s. In the 1990s, his work gave rise to the worldwide first-in-man trial of stem cell gene therapy for the treatment of a rare immunodeficiency (ADA-SCID). His belief in the long-term value of gene therapy prompted him to co-found the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET). He also cofounded the gene therapy company GenEra S.p.A., who later merged into Molmed S.p.A. (a biotech company specializing in innovative cancer therapies) where he served as Director of R&D. He was also involved in setting up the University of Modena's Center for Regenerative Medicine.
Fulvio Mavilio commented that "Généthon is a world-famous benchmark organization that offers unique opportunities in gene therapy for rare diseases. With highly skilled, multidisciplinary research groups and Généthon BioProd (the world largest facility for product gene-based drugs), joining Généthon constitutes an extremely stimulating challenge!".