The prestigious recognition honors a pioneer of lentiviral gene therapy whose discoveries have led to approved treatments for rare diseases, including the first gene therapy brought to the U.S. market through a non-profit development pathway.
Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Full Professor at Vita-Salute San Raffaele University in Milan, has been elected as an International Member of the U.S. National Academy of Medicine (NAM), one of the world's highest honors in health and biomedical science. The Academy recognized him “for pioneering bench-to-bedside development of lentiviral vectors for gene transfer”, technologies that have enabled safe and effective therapies for thousands of patients affected by severe inherited diseases and cancers.
The distinction comes less than a year after a major milestone for the scientific community Naldini helped build. In December 2025, a gene therapy for Wiskott-Aldrich syndrome, a rare disorder of the immune system, was approved by the Food and Drug Administration. Based on the lentiviral platform pioneered at SR-Tiget, the therapy completed a development and regulatory pathway led by Fondazione Telethon, making the Italian charity the first in the world to take a gene therapy from research to U.S. approval.
For Fondazione Telethon and Ospedale San Raffaele, Naldini’s election recognizes both an extraordinary scientific career and a longstanding partnership that has helped transform pioneering research into life-changing treatments for patients with rare genetic diseases.
Under Naldini’s leadership, SR-Tiget has become one of the world’s leading centers for gene and cell therapy research, bringing together discovery science, clinical research and therapeutic development within a unique translational ecosystem. Over the past three decades, his work has helped establish lentiviral vectors as one of the one of the most widely employed tools in biomedical research, whose applications include ex vivo and in vivo gene therapy, cancer immunotherapy and vaccine manufacturing. Of note, 7 out of 18 gene therapy drugs on the EU market and 12 out of 32 on the US market are based on lentiviral vectors.
The Institute's broader scientific legacy includes the development of a gene therapy for metachromatic leukodystrophy, later licensed to Orchard Therapeutics, which helped demonstrate the therapeutic potential of lentiviral gene transfer. SR-Tiget also contributed to the development of the world's first ex vivo stem cell gene therapy, approved in Europe in 2016 for ADA-SCID, for which Fondazione Telethon continues to serve as the organization responsible for making the treatment available to patients in Europe. Together, these achievements reflect a longstanding commitment to translating scientific discoveries into new treatment options for patients with rare genetic diseases.
Today, SR-Tiget brings together more than 300 researchers and professionals from around 20 countries and is internationally recognized as one of the leading hubs for gene and cell therapy research and development. Naldini’s election to the National Academy of Medicine recognizes not only an outstanding scientific career, but also the impact of a shared vision between Fondazione Telethon and Ospedale San Raffaele: advancing pioneering science and ensuring that it ultimately reaches the patients who need it most.
“I am deeply honored to be elected as an International Member of the National Academy of Medicine” said Luigi Naldini.
“I see this recognition as a tribute to the Institute I have the privilege to lead and to the many talented people who, over more than 20 years, have helped bring lentiviral vector-based therapies from the laboratory to patients".
Luigi Naldini
"I am especially grateful to the patients and their families for their trust and participation. We remain committed to advancing these therapies and making them increasingly accessible and sustainable for those who need them”.
