GSK, Fondazione Telethon, and Fondazione San Raffaele Collaborate on Gene Therapy for Rare Diseases

GSK plc has announced a new strategic alliance with Fondazione Telethon and Fondazione San Raffaele to research and develop novel treatments for rare genetic disorders, utilizing ex vivo gene therapy performed on a patient's own bone marrow stem cells.

The collaboration leverages research from the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), a joint venture established in 1995. Under the agreement, GSK gains an exclusive license to develop and commercialize an investigational gene therapy for ADA Severe Combined Immune Deficiency (ADA-SCID), a rare and life-threatening condition affecting approximately 350 children worldwide.

Furthermore, GSK will co-develop six other applications of ex vivo stem cell therapy with Fondazione Telethon and Fondazione San Raffaele. These will utilize a new gene transfer technology developed by HSR-TIGET scientists and hold potential for treating a range of rare disorders, including metachromatic leukodystrophy (MLD) and Wiskott-Aldrich Syndrome (WAS).

Fondazione Telethon will receive an upfront payment of 10 million euros from GSK, with potential for further milestone-based payments. The alliance aims to bring advanced therapies to patients suffering from serious, single-gene disorders.