San Raffaele Telethon Institute for Gene Therapy (SR-Tiget)

Founded in 1996, the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between Fondazione Telethon, a major Italian charity funding research on rare genetic diseases, and Ospedale San Raffaele (OSR), a research hospital of excellence certified by the Ministry of Health. The Institute has been directed by Luigi Naldini since 2008 and is located in Milan (Italy) within the OSR campus, which includes a large multi-disciplinary research hospital, a biomedical research center, the Vita-Salute San Raffaele University and hosts several biotech companies. In its 25-year history, SR-Tiget has provided pioneering and continued contributions to the gene and cell therapy field with relevant discoveries in vector design, gene transfer and gene editing strategies, stem cell biology, identity and mechanism of action of regulatory cells in immune responses and innate immune cells in cancer. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and lysosomal storage disorders, which have already treated 126 patients and led to the filing and marketing approval of 2 novel advanced therapy medicines. More recently, SR-Tiget has also embarked on developing applications of its cell and gene therapy platforms to the treatment of certain types of cancer.