The European Medicines Agency’s CHMP recommended five new medicinal products, including Fondazione Telethon’s ex‑vivo gene therapy Waskyra (etuvetidigene autotemcel) for Wiskott‑Aldrich syndrome. The guidance follows independent assessments and positions the therapy for an approval decision in the EU, a notable outcome for a program shepherded by a non‑profit after being abandoned by multiple companies. Waskyra is the first gene therapy recommended for this rare immunodeficiency and reflects the CHMP’s willingness to endorse treatments developed outside traditional pharma pathways when clinical need is high. The recommendation may accelerate patient access in Europe and set precedents for charity‑driven commercialization routes. Regulators will finalize decisions and labeling, but the CHMP vote shifts momentum toward EU market authorization and highlights evolving collaboration models between charities, academia, and industry in rare‑disease therapeutics.