FDA has cleared Ultragenyx’s one-time AAV8 gene therapy Genglycos (pariglasgene brecaparvovec; DTX-401) for glycogen storage disease type Ia (GSDIa) in patients aged 8 and older. The approval marks the company’s first marketable gene therapy and the first treatment cleared for the underlying cause of the ultra-rare metabolic disorder. Genglycos is designed to deliver a functional gene to the liver to restore the missing enzyme activity that stabilizes blood sugar between meals. Ultragenyx said it plans to have therapy available within 30 to 60 days at specialized treatment centers following the accelerated approval. The clearance comes as Ultragenyx continues navigating prior setbacks in its pipeline and positions the company for commercial ramp-up, including potential monetization of priority review vouchers tied to future regulatory decisions.