A nonprofit organization successfully brought an abandoned rare‑disease gene therapy to market, illustrating alternative models to rescue small or orphan programs that big pharma has shelved. BioCentury coverage described the nonprofit’s role in facilitating market access for the therapy and the potential scalability of the approach for other neglected assets. At the same time, Catalent received two FDA Form 483 observations at Maryland gene‑therapy facilities that manufacture products for companies including Sarepta. The inspection outcomes and subsequent mass layoffs underscore regulatory and manufacturing vulnerabilities in the gene‑therapy supply chain. Together the stories highlight contrasting responses: novel nonprofit commercialization to maintain patient access versus regulatory scrutiny of contract manufacturing capacity needed for clinical and commercial gene therapies.