The FDA approved Waskyra, a gene therapy for Wiskott‑Aldrich syndrome developed by Fondazione Telethon, marking the first regulatory green light for a gene therapy originating from a nonprofit applicant. Pivotal data presented at ASH supported the approval and the agency cited a flexible review tailored to the product’s rare‑disease context. The decision highlights alternative development routes for complex genetic medicines and may spur more academic and nonprofit organizations to pursue translational programs where large pharma has retreated.