The FDA approved Waskyra, the first gene therapy authorized to treat Wiskott–Aldrich syndrome, and the first gene therapy cleared from a nonprofit sponsor. The product, developed by Fondazione Telethon and commercialized under the Waskyra name, was backed by pivotal data presented at the American Society of Hematology showing durable clinical benefit in a rare primary immune‑deficiency. Regulators cited clinical evidence from the pivotal studies presented at ASH during their decision. The authorization marks an unusual pathway: a nonprofit developed the product through academic and translational networks and navigated regulatory and manufacturing hurdles typically handled by commercial developers. Waskyra’s approval underscores ongoing interest in academic‑sourced genetic medicines despite industry pullback from complex one‑time therapies. Companies, payors and policy makers will now confront manufacturing scale, supply‑chain demands and payment models for a therapy approved under an atypical sponsor structure. Clinicians and patient groups will watch rollout plans closely given limited treatment options for severe Wiskott–Aldrich syndrome.