The FDA approved Waskyra, a gene therapy for Wiskott‑Aldrich syndrome developed by a nonprofit foundation, marking the first time an NGO‑led program secured authorization for a genetic medicine. Regulators and company statements highlighted the agency’s flexible review pathways used to clear the therapy and emphasized the program’s clinical evidence package presented at ASH. Stakeholders called the approval historic: it signals alternative development models for rare diseases and may spur further nonprofit or academic translational efforts. Companies and policy groups said the case will be watched for pricing, manufacturing and access precedents for one‑time durable therapies.