A nonprofit organization succeeded in taking an abandoned rare-disease gene therapy to market, reporting a path to patient access where commercial sponsors had exited. The group partnered with Fondazione Telethon and U.S. counterparts to advance a therapy for Wiskott-Aldrich syndrome, shepherding it through development and market entry. BioCentury’s reporting framed the outcome as proof of concept for nonprofit-driven commercialization models. Executives and patient advocates hailed the result as lifesaving for a small patient population, while industry observers noted questions about whether the model can scale across more complex or higher-cost programs.