Ultragenyx announces positive data from its AAV gene therapy DTX401, a phase 1/2 confirmed study group for type Ia glycogenic storage disease

Ultragenyx Pharmaceuticals has announced its adenosic-related virus (AAV)-based gene therapy DTX401, a phase 1/2 study of DTX401 for the treatment of GSDIaCompared to the first two queues, all three patients in queue 3 responded to treatment and showed a rapid decline in demand for cornstarchIn all queues, 100% of patients (n s 9) showed a meaningful and sustained decrease in demand for cornstarch over timeDr Eric Crombez, Chief Medical Officer, Ultragenyx Gene Therapy Research and Development, said: 'In the confirmed third group, continuous glucose monitoring showed early GM expression and the demand for cornstarch decreased faster than in the previous group'GSDIa was the most severe hereditary glycogenstorage diseaseIt is caused by a genetic defect in G6Pase-alpha asse, which prevents the inability to regulate blood sugar (glucose)Hypoglycemia in GSDIa patients can be life-threatening, and the accumulation of complex glycogen in certain organs and tissues can impair the normal functioning of these tissuesIf left untreated for long periods of time, the patient develops severe lactic acidosis, causes renal failure, and may die in infancy or childhoodThere are currently no approved drug therapies for the diseaseAn estimated 6,000 patients worldwide are affected by GSDIaDTX401 is a gene therapy based on AAV8 and is designed to achieve stable expression and activity of G6Pase-alpha under the control of natural promotersDTX401 is administered in the form of a single vein and has been shown in preclinical studies to improve G6Pase-alpha activity and reduce liver glycogen levels, which are good biomarkers for disease developmentDTX401 has been awarded the title of 'Orphan Medicine' in the United States and Europe, and 'Advanced Regenerative Medicine' (RMAT) and 'Fast Track' designation in the United States