The FDA approved Waskyra, a gene therapy for Wiskott‑Aldrich syndrome, marking the first time a non‑profit applicant secured authorization for a genetic medicine. Regulators cited the program’s pivotal data and the nonprofit sponsor’s manufacturing and clinical evidence when granting approval. The decision followed presentations at major hematology meetings and reflects a regulatory willingness to support alternative commercial models for highly specialized therapies. Industry observers described the milestone as a potential model for noncommercial players to shepherd ultra‑rare therapeutics through development and into patients, while raising questions about scale, reimbursement, and long‑term manufacturing sustainability for one‑time gene therapies.