The FDA approved Waskyra, a gene therapy for Wiskott‑Aldrich syndrome, marking the first U.S. authorization of a gene therapy developed and filed by a nonprofit applicant, Fondazione Telethon. The approval follows pivotal data presented at hematology meetings and reflects agency flexibility in evaluating complex genetic medicines from nontraditional sponsors. Regulatory documents and meeting coverage indicate the decision reflects durable clinical benefit in a life‑threatening immune disorder with limited alternatives beyond bone marrow transplant. The nonprofit developer emphasized patient access and the challenges of scaling manufacturing for rare‑disease gene therapies. The approval underscores ongoing shifts: academic and nonprofit groups are increasingly moving into late‑stage genetic‑medicine development as commercial players retreat from some ultra‑rare indications because of manufacturing and reimbursement complexities.