DTX401
for GSDIa
DTX401 (pariglasgene brecaparvovec): Gene therapy for the potential treatment of glycogen storage disease type Ia (GSDIa)
Program overview
Stage: Phase 3
Disease: GSDIa
Prevalence: 6,000 people in commercially accessible geographies
Disease Mechanism: Inability to regulate blood sugar (glucose)
Symptoms: Severe hypoglycemia, seizures, hepatomegaly, growth problems
Treatment Modality: AAV8 gene therapy
Treatment Mechanism: Deliver transgene to produce normally functioning glucose-6-phosphatase
GSDIa: Disorder of energy metabolism leading to inability to maintain normal glucose levels
GSDIa is caused by a defective gene coding for the enzyme G6Pase-α, resulting in the inability to regulate blood sugar (glucose). GSDIa is one type of glycogen storage disorder with the enzymatic deficiency causing the accumulation of glycogen in the liver and kidney. Without the daily use of oral glucose replacement therapy (cornstarch), individuals with GSDIa 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 commercially accessible geographies.
Evaluating DTX401 to establish normal glucose metabolism and reduce or eliminate the need for cornstarch to maintain normal glucose levels
DTX401 (pariglasgene brecaparvovec) is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-α using a single intravenous infusion.
DTX401 has been granted orphan drug designation, regenerative medicine advanced therapy (RMAT) designation and Fast Track designation from the U.S. FDA, as well as PRIority MEdicines (PRIME) and orphan drug designation from the European Medicines Agency.