Ultragenyx will build a new gene therapy manufacturing facility in Bedford

Ultragenyx Pharmaceutical, a US biopharmaceutical company focused on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases, plans to build a new large-scale gene therapy manufacturing facility in Bedford, Massachusetts.

The new facility will enable in-house manufacturing of Ultragenyx’s pipeline of clinical stage adeno-associated virus (AAV)- based gene therapies, including DTX301 for ornithine transcarbamylase (OTC) deficiency (ph I-II), DTX401 for glycogen storage disease type Ia (GSDIa) (ph I-II), and UX701 for Wilson disease (IND Submission), as well as other preclinical programs. This portfolio is also set to enlarge since Ultragenyx and Solid Biosciences has announced last month a strategic collaboration and license agreement to focus on the development and commercialization of new gene therapies for Duchenne muscular dystrophy. Ultragenyx intends to use its AAV-based HeLa PCL platform including HeLa 3.0 improvements for the development of these product candidates. The capsid planned is an AAV8 variant with a favorable immunological profile that has been used successfully in the large scale 2,000 liter production process.

A new facility to support pipeline advancement

Ultragenyx will continue to leverage some contract manufacturing organizations in addition to its own manufacturing facility. The company 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. “ 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 planned Phase I facility will encompass 100,000 square feet (9200 m²) 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.

Anne-Lise Berthier (09/11/2020)