After being dropped by two commercial partners, Fondazione Telethon shepherded Waskyra (etuvetidigene autotemcel) — an ex‑vivo autologous hematopoietic stem‑cell gene therapy for Wiskott‑Aldrich syndrome — back to regulatory view and secured an EMA recommendation. The charity’s involvement bridges a commercialization gap left by pharma partners and moves the therapy closer to European market authorization for a rare, life‑threatening primary immunodeficiency. The case demonstrates an alternative commercialization path where non‑profit actors advance late‑stage, high‑value biologics that large firms deem non‑strategic. Regulators, payers, and manufacturers will watch pricing, manufacturing scale‑up and long‑term follow‑up commitments as Waskyra transitions toward patients.