RARE Daily

Ultragenyx Plans Large-scale Gene Therapy Manufacturing Facility to Support Rare Disease Pipeline

November 9, 2020

Rare Daily Staff

Ultragenyx Pharmaceutical announced that it plans to build a large-scale gene therapy manufacturing facility in Bedford, Massachusetts to enable in-house manufacturing of its pipeline of AAV-based gene therapies.

The move will give Ultragenyx more control on production, a major hurdle for gene therapy drug developers as more companies move into the growing space and access to manufacturing capacity becomes a mounting issue.

Ultragenyx’s pipeline includes gene therapies in development for the treatment of ornithine transcarbamylase (OTC) deficiency, glycogen storage disease type Ia (GSDIa), and Wilson disease, as well as other preclinical programs. It will continue to leverage some contract manufacturing organizations in addition to its own manufacturing facility.

The new facility will house both of Ultragenyx’s gene therapy manufacturing platforms: the HeLa producer cell line (PCL) platform, a highly scalable platform that enables large 2,000 liter commercial-scale manufacturing and yields reproducible high-quality product, 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,” 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 planned phase 1 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 2 expansion on-site that could be used to double capacity if needed.

Photo: Dennis Huang, chief technical operations officer at Ultragenyx

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