DTX 401 is an investigational AAV gene therapy being developed for the potential treatment of patients with Glycogen Storage Disease Type 1a (GSDIa). DTX 401 is designed to address 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 GSDIa 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.
GSD1a has completed preclinical studies.