AUG 23 2026
Ultragenyx Pharmaceutical Inc. — DTX401 $RARE
for glycogen storage disease type Ia
About
Glycogen storage disease type Ia is a serious metabolic disorder where the liver cannot properly release stored sugar into the blood, forcing patients to constantly consume glucose to prevent life-threatening low blood sugar. DTX401 is a gene therapy designed to deliver a functional copy of the enzyme the liver lacks. By restoring the liver's natural ability to manage blood sugar, this treatment could potentially reduce or eliminate the need for round-the-clock glucose supplements.
Pivotal trial
The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose control.
Source: ClinicalTrials.gov
Also in development
Drug Watch — recent news on DTX401
- Rolling BLA submission for DTX401 (pariglasgene brecaparvovec) AAV gene therapy in Glycogen Storage Disease Type Ia: Phase 3 data show reduced daily cornstarch intake while maintaining low hypoglycemia levels - Vera Health
- Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa) - finance.yahoo.com
- Ultragenyx Finishes Rolling Submission of BLA for Glycogen Storage Disease Gene Therapy DTX401 - CGTLive®