DTX 401 for GSD1a

DTX 401 is an investigational AAV gene therapy being developed for the potential treatment of patients with Glycogen Storage Disease Type 1a (GSD1a). DTX 401 is designed to addres the defective gene for the enzyme glucose-6-phosphatase-α, a defect that results in the inability to regulate blood sugar (glucose).

GSD1a is the most common genetically inherited glycogen storage disease. Hypoglycemia in GSD1a patients can be life-threatening, while the accumulation of the complex sugar glycogen in certain organs and tissues can impair the ability of these tissues to function normally. If chronically untreated, patients develop severe lactic acidosis, can progress to renal failure, and die in infancy or childhood. There are no approved pharmacological therapies. An estimated 6,000 or more patients are affected by GSDIa worldwide.

Clinical Trials

GSD1a has completed preclinical studies.

DTX 401 for GSD1a

Stage

Preclinical

Disease

Glycogen Storage Disease Type Ia (GSDIa).

Prevalence

6,000 developed world patients

Disease mechanism

Inability to regulate blood sugar (glucose)

Symptoms

Severe hypoglycemia and hepatomegaly

Treatment mechanism

Deliver glucose-6-phosphatase