Ultragenyx opened a new gene therapy manufacturing facility for clinical and commercial-stage adeno-associated virus (AAV)-based gene therapy treatments in Bedford, Massachusetts. Credit: Ultragenyx Pharmaceutical Inc.
The new facility is located on a 10.7-acre site, covering 110,000ft² area. Credit: Ultragenyx Pharmaceutical Inc.
The facility utilises Pinnacle PCL™ manufacturing technology. Credit: Ultragenyx Pharmaceutical Inc.
Ultragenyx plans to recruit 120 people across a broad spectrum of skillsets and backgrounds for the new facility. Credit: Ultragenyx Pharmaceutical Inc.

Ultragenyx Pharmaceutical, a biopharmaceutical company based in the US, opened its first large-scale gene therapy manufacturing facility in Bedford, Massachusetts, in June 2023.

The facility allows the company to carry out uninterrupted and streamlined in-house production of adeno-associated virus (AAV)-based gene therapy treatments at both clinical and commercial stages.

Ultragenyx previously relied on contracted pharmaceutical manufacturing facilities to manufacture the gene therapy products in its pipeline. The new facility integrated the company’s gene therapy research, development and manufacturing, and expedited the process, ensuring that it can promptly address the pressing requirements of patients with rare and ultra-rare diseases. The company is also expected to maintain a certain level of contracted manufacturing.

The development of the facility was supported by the town of Bedford and the Commonwealth of Massachusetts. The company intends to recruit 120 people across a broad spectrum of skill sets and backgrounds to staff the facility, adding to its existing workforce, including 350 employees in Bedford, Woburn, and Cambridge.


The facility is located on a 10.7-acre site at 170 Middlesex Turnpike, Bedford.

Bedford was selected because of its proximity to the company’s Greater Boston research and development operations. The facility also strengthens the life sciences cluster in Bedford.

Ultragenyx’s new gene therapy manufacturing facility details

The state-of-the-art facility is built on a 110,000ft2 area for manufacturing gene therapy products using a proprietary Pinnacle producer cell line (PCL) manufacturing platform.

The Pinnacle PCL platform is a clonal AAV manufacturing system engineered to consistently generate high yields of viable, unaltered AAV vectors while maximising the manufacturing of entire AAV capsids. The cells are derived from the modified HeLa cells.

PCL is developed to improve the safety and production of AAV gene therapy, requiring comparatively greater dosages, such as those affecting the central nervous system and muscular tissues. It increases product performance and yield, simultaneously lowering costs and accelerating manufacturing.

The new facility uses both the company’s gene therapy production platforms, HeLa PCL, and the HEK293 transient transfection manufacturing technology platform. The HeLa PCL platform allows production at a large commercial scale of 2,000l for high-quality products. The HEK293 transient transfection system is also used for AAV manufacturing.

Ultragenyx’s new gene therapy manufacturing facility design

The first floor of the facility is equipped with two separate bulk drug substance manufacturing suites with a production capacity of 30 runs per year, high-bay warehouse storage and double-height lobby space. The second floor features a single drug product suite, raw materials, electron microscopy laboratories and spaces to support multi-function collaboration, meetings, conferences and offices.

The two levels are separated by a ceiling that can be walked on, located above the manufacturing equipment platform. A vertical amenity cafe connects the facility.

A 15,000ft2 modular mechanical penthouse is at the centre of the roof. On the east side of the building is a utility yard. The modular penthouse fully embraces the integrated project delivery philosophy. It contains the facility’s custom current good manufacturing practice air-handling units and boilers and its humidification, steam and glycol recovery system. The self-supporting structural enclosure also houses variable frequency drives and supporting electrical equipment.

Sustainable features of the facility include natural light and connection to the surrounding woodlands and forests.

Ultragenyx has obtained land and development rights for an additional 86,000ft2 of expansion space, which can be utilised if needed to double the current on-site capacity.

Products manufactured at the facility

The facility manufactures the company’s 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.

Ultragenyx’s partnerships in gene therapy

Ultragenyx signed a strategic non-exclusive licence and technology access agreement with Daiichi Sankyo, a Japanese healthcare company, for Ultragenyx’s proprietary AAV-based gene therapy manufacturing technologies in March 2020.

Another agreement was signed between Ultragenyx and Solid Biosciences, a  biotech company based in the US, to develop and commercialise new gene therapies for Duchenne muscular dystrophy combining Solid’s differentiated microdystrophin construct, Ultragenyx’s HeLa PCL manufacturing platform, and AAV8 variants, in October 2020.

German pharmaceutical company Bayer utilises the company’s PCL platform to produce its haemophilia A gene therapy at a 2,000l scale.

Contractors involved

CRB Group, a construction and consulting company based in the US, was responsible for the design and engineering of the facility.

Gaston Electrical Services, an electrical services provider based in the US, supplied full core and shell electrical services for the facility, under a subcontract awarded by the Cardinal Group, a US-based construction and project management company.