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Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting
Oral presentations include longer term durability data from DTX301 and DTX401 and showcase the Pinnacle PCL™ (AAV vector producer cell line) platform and
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Oral presentations include longer term durability data from DTX301 and DTX401 and showcase the Pinnacle PCL™ (AAV vector producer cell line) platform and technology\nNOVATO, Calif., May 08, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultrarare diseases, today announced that clinical, preclinical and manufacturing data from its investigational gene therapy programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, which will be held May 16-20, 2023 at the Los Angeles Convention Center in Los Angeles, California. The company will present additional data supporting its gene therapy portfolio and discuss critical topics during the clinical trials spotlight symposium and at pre-meeting workshops. Clinical and pre-clinical presentations: Oral presentation: Efficacy and safety at week 52 and up to four years in adults with glycogen storage disease type Ia (GSDIa): Results from a Phase 1/2 clinical trial and long-term follow-up study of DTX401, an AAV-8-mediated, liver-directed gene therapy (Abstract #4) Date/Time: Thursday, May 18, 8:00 – 8:15 AM PDTLocation: Concourse Hall 152 and 153First author: Rebecca Riba-Wolman, M.D., University of Connecticut Oral presentation: Long-term Safety and Efficacy of DTX301 in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: A Phase 1/2 Trial (Abstract #128) Date/Time: Thursday, May 18, 2:45 PM - 3:00 PM PDTLocation: Room 403 ABPresenter: Cary Harding, M.D., Oregon Health & Science University Poster presentation: Immunosuppression to Inhibit Capsid-Specific Humoral Immune Responses in High-dose AAV Gene Therapy in Cynomolgus Macaques (Board No. 1191) Date/Time: Thursday, May 18, 12:00 PM PDTPresenter: Deniz Erturk-Hasdemir, Ph.D., Ultragenyx Additional presentations including the company’s manufacturing capabilities and Pinnacle PCL™ (AAV vector producer cell line) platform and technology: Oral presentation: In Vivo and In Vitro Assessment of Residual DNA Impurity-Derived Transcriptions (Abstract #109) Date/Time: Thursday, May 18, 1:30 – 1:45 PM PDTLocation: Concourse Hall 150 and 151Presenter: Hsin-I Jen, Ph.D., Ultragenyx Poster presentation: Early Development of In Vitro Potency Assays for Rare and Ultr...