Naldini, the pioneer of gene therapy, joins the pantheon of US medicine
The director of the Telethon Institute for Gene Therapy has been elected an international member of the National Academy of Medicine: recognition for the research that has brought treatments to thousands of patients
Key points
An accolade that recognises thirty years of research and establishes the Milanese school of gene therapy on the world stage. Luigi Naldini, director of the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and full professor at the Vita-Salute San Raffaele University, has been elected an international member of the US National Academy of Medicine (NAM), one of the world’s most authoritative institutions in the field of health and biomedical sciences.
The Academy cited Naldini’s pioneering contribution to the development and clinical application of lentiviral vectors for gene transfer as the reason for his appointment; this technology has made safe and effective treatments possible for thousands of people with serious inherited genetic disorders and cancers.
The Wiskott-Aldrich finish line
This election comes less than a year after a historic achievement for the scientific community that Naldini helped to build. In December 2025, the US Food and Drug Administration approved a gene therapy for Wiskott-Aldrich syndrome, a rare immune system disorder. The treatment is based on the lentiviral platform developed at SR-Tiget and underwent a development and authorisation process led by the Telethon Foundation, which has thus become the first non-profit organisation in the world to take a gene therapy from research to approval in the US.
A world-leading centre
Under Naldini’s leadership, SR-Tiget has become one of the leading international centres for research into gene and cell therapy, integrating basic research, clinical research and therapeutic development into a single translational ecosystem. Over the past thirty years, lentiviral vectors have established themselves as one of the most widely used tools in biomedical research, with applications ranging from ex vivo and in vivo gene therapy to cancer immunotherapy and vaccine production. The figures illustrate the scale of this impact: 7 of the 18 gene therapies approved in the European Union and 12 of the 32 approved in the United States are based on lentiviral vectors.
The Institute’s achievements also include gene therapy for metachromatic leukodystrophy, which was subsequently licensed to Orchard Therapeutics. SR-Tiget also developed the first ex vivo gene therapy using stem cells to be approved in Europe, in 2016, for ADA-SCID: the Telethon Foundation continues to ensure its availability to European patients.
