The FDA approved Waskyra, a gene therapy for Wiskott‑Aldrich syndrome, marking the first time the agency has cleared a genetic medicine developed and submitted by a nonprofit sponsor, Fondazione Telethon. Pivotal data were presented at the American Society of Hematology and supported the approval for this rare blood-and-immune disorder that typically requires bone marrow transplant. Regulators and industry view the approval as proof that non-traditional developers can advance complex genetic medicines through the clinic when supported by rigorous evidence and manufacturing plans. STAT and MedCity News noted the decision amid broader pressures on the gene‑therapy field—high costs, manufacturing complexity and sparse commercial returns—that have driven many companies away from the space. The milestone may inform future regulatory and financing models for academic and charitable sponsors advancing one‑time genetic medicines for rare diseases. It also underscores the FDA’s willingness to consider diverse applicants when data and quality systems meet agency expectations.