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BioWorld - Thursday, January 8, 2026
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Home » Ultragenyx gene therapy hits phase III goal in rare disease GSD1a
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Ultragenyx gene therapy hits phase III goal in rare disease GSD1a

May 31, 2024
By Jennifer Boggs
Ultragenyx Pharmaceutical Inc. anticipates a meeting later this year with the U.S. FDA to discuss a BLA filing for gene therapy DTX-401 as the first potential medical treatment for glycogen storage disease type 1a (GSD1a) after the phase III study hit its primary endpoint and two key secondary endpoints.
BioWorld Clinical Endocrine/metabolic Gene therapy U.S. FDA

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