Business
Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting
Oral presentations include longer term durability data from DTX301 and DTX401, and manufacturing data on the Pinnacle PCL™ (AAV vector Producer Cell Line)
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Oral presentations include longer term durability data from DTX301 and DTX401, and manufacturing data on the Pinnacle PCL™ (AAV vector Producer Cell Line) platform and technology\nNOVATO, Calif., May 09, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare 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) 25th Annual Meeting, which will be held both in person and virtually May 16-19, 2022. The company will present new data supporting its gene therapy portfolio and discuss critical topics during a scientific symposium on Accelerated Approval and at the pre-meeting workshops. Clinical and pre-clinical presentations include: Oral presentation: Safety and Efficacy of DTX301 in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: A Phase 1/2 Trial (Abstract #463) Date/Time: Tuesday, May 17, 4:15-4:30 PM ETPresenter: Cary Harding, M.D., Oregon Health & Science University Oral presentation: Sustained Efficacy and Safety at Week 52 and up to Three Years in Adults with Glycogen Storage Disease Type Ia (GSDIa): Results from a Phase 1/2 Clinical Trial of DTX401, an AAV8-mediated, Liver-directed Gene Therapy (Abstract #1212) Date/Time: Thursday, May 19, 11:00-11:15 AM ETPresenter: Rebecca Riba-Wolman, M.D., University of Connecticut Poster presentation: Improving Neuronal Gene Transfer to the Brain After CSF Administration of AAV9 in Juvenile Non-Human Primates (Board No. Tu-143) Date/Time: Tuesday, May 17, 5:30-6:30 PM ETPresenter: Maggie Wright, Ph.D, Ultragenyx Poster presentation: Understanding the Educational Needs of United States Physicians Related to Gene Therapy (Board No. Tu-67) Date/Time: Tuesday, May 17, 5:30-6:30 PM ETPresenter: Emily Belcher, CE Outcomes Manufacturing presentations covering the company’s Pinnacle PCL™ (AAV vector Producer Cell Line) platform and technology include: Oral presentation: Development and Characterization of Highly Optimized Monoclonal Producer Cell Lines (PCLs) for the Treatment of CDKL5 Deficiency Disorder (CDD) (Abstract #863) Date/Time: Wednesday, May 18, 4:15-4:30 PM ETPresenter: Laurie Tran, MSc, Ult...