Solving-The-Hardest-Problems-Through-Partnership-And-Innovation-Part-1

A researcher from the San Raffaele Telethon Institute for Gene Therapy presents the institute's 30-year journey from foundational science to three approved hematopoietic stem cell gene therapies for ADA-SCID, metachromatic leukodystrophy, and Wiskott-Aldrich syndrome. The talk traces strategic partnerships with GSK and Orchard Therapeutics, and Fondazione Telethon's unprecedented role as a non-profit commercializer after industry partners withdrew for commercial reasons. The speaker closes by identifying persistent ATMP development bottlenecks and calling for deeper cross-sector collaboration through initiatives like Joint for ATMP and Agora.

Key Takeaways

• Three hematopoietic stem cell gene therapies from the San Raffaele/Telethon program are approved as of 2026 (ADA-SCID, MLD, Wiskott-Aldrich syndrome), with ADA-SCID being the first such therapy ever approved globally and all 50 treated patients still alive with durable benefit at 10-15+ years.
• Fondazione Telethon became the first charity to complete the full marketing authorization pathway for gene therapies, taking over commercial licenses for ADA-SCID and Wiskott-Aldrich syndrome when industry partners discontinued marketing for commercial reasons.
• The 2010 GSK alliance model—academia contributing science and patient data, industry contributing GMP, regulatory, and commercial expertise—enabled progression from Phase 1 to market approval across five rare disease programs.
• Regulatory approvals were achieved with very small cohorts (18 patients for ADA-SCID, fewer than 30 for MLD and Wiskott-Aldrich syndrome), provided clinical evidence is robust and regulatory authorities are engaged early in development.
• Persistent ATMP development bottlenecks—including difficulty reusing clinical data and reintroducing innovation—remain unresolved; EU initiatives like Joint for ATMP and Agora are working to propose systemic solutions to policymakers and regulators.