Ultragenyx's gene therapy wins first FDA approval for rare metabolic disorder

Ultragenyx's gene therapy Genglycos received FDA approval to treat glycogen storage disease type Ia, a rare metabolic disorder, providing a novel treatment that targets the disease's root cause and could reduce patients' reliance on cornstarch therapy.

This Wire brief sits within Fusion42's coverage of Biotech, and 6 sources have reported it between 19 Aug 2026 and 22 Aug 2026.