Ultragenyx announced a workforce reduction of about 130 people (roughly 10%) amid restructuring intended to reach profitability by 2027. The company reported that the FDA again delayed approval for its gene therapy UX111 for Sanfilippo syndrome type A, requesting additional manufacturing documentation after an earlier deficiency cited in 2025. Executives told investors the layoffs and expense reductions will preserve runway and concentrate resources on highest-value programs. Analysts noted the combination of late-stage trial failures and unresolved manufacturing questions for gene therapies highlights regulatory and manufacturing hurdles that can materially affect small rare-disease biotechs’ commercial trajectories.