Fondazione Telethon

Overview

Italian not-for-profit charity that co-founded TIGET with San Raffaele Hospital, funded rare disease gene therapy research, and became the first charity to achieve full marketing authorization for gene therapy products in both the US and EU. Fondazione Telethon co-founded the San Raffaele Telethon Institute for Gene Therapy approximately 30 years before 2026, partnering with San Raffaele Hospital to support research into rare genetic diseases. Fondazione Telethon took over the ADA-SCID gene therapy license after the commercial company stopped marketing the product for purely commercial reasons, becoming the first non-profit entity to maintain an approved gene therapy in the market. (Segment 1 (00:00-05:00)) Telaton Foundation, a not-for-profit charity, took over the San Raffaele gene therapy program and received regulatory approvals for gene therapy products in both the US and Europe, creating a model for academic-to-commercial translation outside the pharma industry. After receiving US and European approvals, Telaton Foundation received many collaboration requests from patient associations for hundreds of other diseases, illustrating that the not-for-profit model cannot scale to meet demand without alternative funding models. (Segment 4-5 (20:00-25:00))