Benzinga | Nov 29, 2021 08:06AM EST

Ultragenyx Pharmaceutical Announces Additional Multi-Year Durability Data From Phase 1/2 AAV Gene Therapy Studies

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced new long-term durability data from the Phase 1/2 studies of DTX401 for Glycogen Storage Disease Type Ia (GSDIa) and DTX301 for Ornithine Transcarbamylase (OTC) deficiency that were presented at the 14th International Congress of Inborn Errors of Metabolism (ICIEM).



"With initial patients now in year 3 following dosing with DTX401 and DTX301 we are seeing the impact of long-term, durable biological responses and the establishment of normal metabolic pathways to allow for the release of glucose from glycogen during periods of fasting for patients with GSDIa and the breakdown of ammonia for patients with OTC deficiency," said Eric Crombez, M.D., Chief Medical Officer, Ultragenyx Gene Therapy and Inborn Errors of Metabolism. "By directly correcting the underlying genetic cause of disease these gene therapies have the potential to provide meaningful quality-of-life improvements for patients and enable new flexibility and independence to take part in a more active and less-restrictive lifestyle."