The FDA approved Waskyra, a gene therapy for Wiskott‑Aldrich syndrome, marking the first FDA authorization of a gene therapy developed and filed by a nonprofit sponsor. Regulators highlighted flexible application of review standards in granting the approval; pivotal data were presented at the American Society of Hematology meeting. Fondazione Telethon led development and submitted clinical data showing durable benefit in this rare combined immunodeficiency. Regulators and the nonprofit said the approval underscores alternative development paths for complex genetic medicines and could influence how academic and nonprofit groups advance costly one‑time therapies. Sources: MedCity News coverage of the FDA approval and STAT reporting on the nonprofit milestone.