DTX401: AAV8 Gene Therapy for Glycogen Storage Disease Type Ia (GSDla)
GSDIa: the most severe genetically inherited glycogen storage disease
GSDIa is caused by a defective gene for the enzyme glucose-6-phosphatase (G6Pase). As a result, the body is unable to regulate glucose (blood sugar), and the complex sugar glycogen can accumulate in certain organs and tissues, impairing their ability to function normally. Without chronic use of oral glucose replacement therapy (cornstarch), patients become severely hypoglycemic, which can be life-threatening. They also can develop severe lactic acidosis and progress to renal failure. No pharmacologic therapies are approved for GSDIa, which affects an estimated 6,000 people in the developed world.
Evaluating DTX401 to improve glucose metabolism and reduce or eliminate the need for cornstarch
DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-α using a single intravenous infusion. In an ongoing Phase 1/2 clinical study, all nine patients showed a clinical response, with significant reductions in the need for cornstarch and improvements in glucose control and other metabolic parameters compared to baseline. A Phase 3 clinical study is planned to evaluate the ability of DTX401 to reduce the use of cornstarch while maintaining glucose control.
DTX401 was granted Orphan Drug Designation in the United States, EU and United Kingdom, and Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation in the United States.
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