Ultragenyx won accelerated FDA approval for Genglycos (pariglasgene brecaparvovec; DTX-401), marking the first approved treatment for glycogen storage disease type Ia (GSDIa). The AAV8 gene therapy is authorized for patients age 8 and older and targets the underlying enzyme deficiency driving life-threatening blood glucose instability between meals. Ultragenyx said the therapy is a one-time treatment delivered to the liver to restore enzyme function. The approval expands the company’s approved-product base and gives it its first marketable gene therapy asset. Ultragenyx also communicated plans for therapy availability at specialized treatment centers within weeks of approval. Analysts framed the decision as a material step toward profitability, with management also referencing the potential value of a priority review voucher strategy and other regulatory timelines for additional gene-therapy candidates.