Pharma Focus Asia

Ultragenyx Announces Plans to Build Large-scale Gene Therapy Manufacturing Facility to Support Pipeline of Therapies for Rare Diseases

Tuesday, November 10, 2020

Ultragenyx Pharmaceutical Inc., 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 leader in the life sciences because of our talented workforce, our world-class companies and institutions, and our embrace of public-private collaboration. We welcome Ultragenyx and their important work to our ecosystem.”

“We are thrilled that Ultragenyx chose Bedford as the location of its new state-of-the-art bio-manufacturing facility, which further strengthens Bedford’s life science cluster,” said Sarah Stanton, Bedford Town Manager. “We appreciate Ultragenyx’s significant investment in our community bringing jobs and economic benefits to Bedford and the region, and look forward to a long-term partnership with Ultragenyx.”

The planned Phase I facility will encompass 100,000 square feet and provide important internal capacity to develop and manufacture supply of Ultragenyx’s gene therapies for both clinical stage and approved products. The facility will be able to support two independent manufacturing suites with a capacity of 30 runs per year. Construction of the new facility has begun and is expected to be complete in 2023. As the facility becomes fully operational, Ultragenyx expects to hire approximately 100 to 150 full-time employees, over a five-year period across a broad range of functions and skill sets, adding to their existing base of employees in Massachusetts. Ultragenyx already controls land and development rights for an additional 86,000 square feet of Phase II expansion on-site which could be used to double capacity if needed.

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