The FDA approved Waskyra, a gene therapy for Wiskott‑Aldrich syndrome developed and sponsored by a nonprofit foundation, marking the first gene therapy authorization awarded to a non‑commercial applicant. Regulatory documents and press coverage note the agency exercised flexible pathways and early engagement to reach a decision for this rare immune disorder. Advocates hailed the approval as a proof of concept for alternative developers—academic groups and charities—to shepherd complex biologics into the clinic and to market. Industry analysts said the approval could catalyze new academic commercialization models but underscored the long‑term manufacturing and reimbursement challenges that accompany one‑time gene therapies. The decision also refocuses attention on FDA review frameworks for individualized and small‑population genetic medicines, and will be closely watched by sponsors pursuing similar orphan or n‑of‑1 programs.