Ultragenyx received FDA accelerated approval for Genglycos (pariglasgene brecaparvovec; DTX-401), an AAV8 gene therapy for glycogen storage disease type Ia (GSDIa) in patients age 8 and older. The decision is designed to address the underlying enzymatic deficiency that disrupts blood sugar stabilization between meals. The therapy is a one-time treatment intended to deliver a functional gene to the liver. Ultragenyx’s approval adds to its growing platform in rare metabolic disease and marks its first marketable gene therapy after multiple earlier program setbacks. Company disclosures put the wholesale acquisition cost at $2.7 million per patient, with plans to make the therapy available within 30 to 60 days at specialized treatment centers. Ultragenyx also highlighted that the approval comes with a priority review voucher plan. Investors and analysts are now likely to focus on how the company balances commercialization of Genglycos with late-stage milestones across its gene therapy pipeline, including upcoming FDA actions on other rare-disease assets.