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Ultragenyx Announces Positive Longer-term Durability Data from Two Phase 1/2 Gene Therapy Studies at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting
Durable and clinically meaningful responses sustained for more than three years post-treatment in study of DTX401 for GSDIa and four or more years in study of
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Durable and clinically meaningful responses sustained for more than three years post-treatment in study of DTX401 for GSDIa and four or more years in study of DTX301 for OTC NOVATO, Calif., May 19, 2022 (GLOBE NEWSWIRE) -- 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 positive longer-term efficacy and safety data from Phase 1/2 studies of DTX401 for the potential treatment of Glycogen Storage Disease Type Ia (GSDIa) and DTX301 for the potential treatment of Ornithine Transcarbamylase (OTC) deficiency. Results demonstrate ongoing durability of response and safety for both gene therapy programs as of the data cutoff date. Data were presented this week at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting. “We are encouraged by the longer-term durable responses and metabolic stability we continue to observe in our lead gene therapy programs and the progress we’re making toward bringing these investigational gene therapies to patients with GSDIa and OTC,” said Eric Crombez, M.D., Chief Medical Officer, Ultragenyx Gene Therapy and Inborn Errors of Metabolism. “The potential to establish normal metabolism with gene therapy could allow these patients to live without restrictive or burdensome diets and medications and lead to increased quality of life.” DTX401 (GSDIa) Phase 1/2 UpdateLonger-term Phase 1/2 data demonstrate durability of response, with sustained responses lasting more than 3.5 years since treatmentAll 12 patients in the study continue to demonstrate improved glucose control, experiencing reductions in oral glucose replacement with cornstarch. Overall, patients reached a mean total daily reduction of cornstarch intake of 70% (p-value","length":2140,"tagName":"div"}]