A nonprofit effort brought an orphaned rare‑disease gene therapy to market by rescuing development and facilitating access, according to BioCentury’s reporting. The case involved Fondazione Telethon partnering with a U.S. nonprofit to advance a therapy for Wiskott‑Aldrich syndrome. The story details the operational, regulatory and commercialization challenges nonprofits face when moving complex biologics to patients and raises questions about whether the model can be replicated at scale for other neglected therapies.