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The company is one of the first in the U.S. with complete end-to-end gene therapy capabilities in Massachusetts, from research and development to therapeutic production at scale

BEDFORD, Mass., June 21, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today officially opened its gene therapy manufacturing facility in Bedford, Massachusetts. The company has a large and diverse clinical pipeline of late-stage gene therapies for the potential treatment of rare genetic conditions. This new, state-of-the-art facility will enable Ultragenyx to produce gene therapy treatments reliably and at scale, leveraging the company’s proprietary Pinnacle PCL^TM (producer cell line) manufacturing platform. Pinnacle PCL enables greater efficiency in producing adeno-associated virus (AAV) gene therapies with improved product quality and yield, lower cost and increased speed of production.

“Today we’re celebrating the result of several years’ worth of effort by hundreds of people, all driven by a single shared goal – to transform the lives of people living with rare disease,” said Dennis Huang, chief technical operations officer and executive vice president for gene therapy research and development at Ultragenyx. “Through this facility, Ultragenyx will develop and produce gene therapy treatments at greater scale than previously possible, which we believe will make these rare disease medicines more accessible to the people who need them.”

Ultragenyx recently finished construction of the 110,000-square-foot gene therapy manufacturing facility on a 10.7-acre site in Bedford, which was selected due to its proximity to Ultragenyx’s research and development operations in the Greater Boston area. The company plans to hire 120 employees to staff the facility from a range of skill levels and backgrounds. Ultragenyx currently employs more than 350 people in Massachusetts across its sites in Bedford, Woburn and Cambridge – more than a quarter of its global workforce.

The company worked closely with the Town of Bedford and various community partners in the region, starting with site selection and groundbreaking and continuing through construction and activation. Throughout the process, Ultragenyx developed close working partnerships with the Town of Bedford, local educational institutions including Middlesex Community College, leading life science organizations such as Massachusetts Biotechnology Council (MassBio) and Massachusetts Life Sciences Center and organizations including the Bedford and Woburn Chambers of Commerce and Middlesex 3 Coalition, among others. Ultragenyx is committed to continued collaboration with these local and regional partners moving forward.

Ultragenyx will hold a Grand Opening ceremony to celebrate with all its Massachusetts-based employees and invited guests on Wednesday, June 21 from 1:00pm to 2:00pm EDT. The event will include remarks from Ultragenyx leaders, local elected officials and rare disease patient advocates.

About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/7059e879-4e18-468b-880e-886b2923ff24

Contacts:

Investors
Joshua Higa
ir@ultragenyx.com

Media
Jeff Blake
415-612-7784
media@ultragenyx.com