The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended five new products, including a gene therapy developed by Fondazione Telethon for Wiskott‑Aldrich syndrome. The endorsement signals regulatory momentum for rare‑disease gene therapies backed by non‑profit initiatives. Waskyra (etuvetidigene autotemcel), an ex‑vivo HSC gene therapy for WAS, reached CHMP consideration after being advanced by a charity following prior commercial partners’ exits. The recommendation underscores alternative routes to commercialization for niche gene therapies. Regulators and payers across Europe will now evaluate benefit‑risk and reimbursement; the CHMP’s recommendations may prompt other small developers and charities to pursue EMA pathways for rare‑disease programs.