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Ultragenyx Announces Plans to Build Large-scale Gene Therapy Manufacturing Facility to Support Pipeline of Therapies for Rare Diseases
State-of-the-art facility to be located in Bedford, Massachusetts NOVATO, Calif., Nov. 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"State-of-the-art facility to be located in Bedford, Massachusetts\n NOVATO, Calif., Nov. 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare genetic diseases, today announced that it plans to build a new large-scale gene therapy manufacturing facility in Bedford, Massachusetts. The new facility will enable in-house manufacturing of the Company’s pipeline of clinical stage adeno-associated virus (AAV)- based gene therapies, including DTX301 for ornithine transcarbamylase (OTC) deficiency, DTX401 for glycogen storage disease type Ia (GSDIa), and UX701 for Wilson disease, as well as other preclinical programs. The company will continue to leverage some contract manufacturing organizations in addition to its own manufacturing facility. Ultragenyx will use both of its gene therapy manufacturing platforms at the new facility: the HeLa producer cell line (PCL) platform which enables large 2,000 liter commercial-scale manufacturing and yields high-quality product from a highly reproducible, highly scalable platform, and the Company’s HEK293 transient transfection system. “We believe that gene therapy is the optimal way to treat many rare and ultra-rare genetic diseases. Our decision to build a state-of-the art manufacturing facility is the logical next step for us as we advance our two clinical-stage programs toward Phase 3 studies, our Wilson Disease program toward IND later this year and make progress in both our HeLa PCL and HEK293 transient transfection manufacturing technology platforms,” said Dennis Huang, Chief Technical Operations Officer at Ultragenyx. “Developing internal manufacturing capabilities will allow us to enhance production processes, enabling us to further optimize quality and scale and ultimately reduce the time it takes to bring our gene therapy solutions to patients.” “The Baker-Polito Administration is thrilled that Ultragenyx Pharmaceutical has chosen Massachusetts as home for its new manufacturing facility, bringing new jobs to an already strong life sciences cluster in Bedford,” said Secretary of Housing and Economic Development Mike Kennealy, who also serves as co-chair of the Massachusetts Life Sciences Center’s Board of Directors. “The Commonwealth remains a global ...