Core Insights - Ultragenyx Pharmaceutical Inc. has completed the rolling submission of its Biologics License Application (BLA) for DTX401, an AAV gene therapy aimed at treating Glycogen Storage Disease Type Ia (GSDIa) [1][2] - If approved, DTX401 will be the first therapy to address the underlying cause of GSDIa, which currently has no approved pharmacologic treatments [5] Company Overview - Ultragenyx is a biopharmaceutical company focused on developing novel therapies for serious rare and ultra-rare genetic diseases, with a portfolio aimed at addressing high unmet medical needs [6][7] - The company has received multiple designations from the FDA for DTX401, including Rare Pediatric Disease designation, orphan drug designation, Fast Track designation, and regenerative medicine advanced therapy (RMAT) designation [4] Clinical Development - The BLA submission for DTX401 is based on data from a clinical program involving 52 treated patients and up to six years of follow-up, demonstrating significant reductions in daily cornstarch intake and improved patient quality of life [2][3] - DTX401 has shown to improve G6Pase activity and reduce hepatic glycogen levels, which are critical for managing glucose metabolism in patients with GSDIa [4] Disease Context - GSDIa is a rare and life-threatening condition caused by mutations in the G6PC gene, leading to severe hypoglycemia and other complications [5] - The disease affects approximately 6,000 individuals in commercially accessible regions, highlighting the need for effective treatment options [5]