Ultragenyx: MA plant to support AAV gene therapy pipeline

Ultragenyx has opened a manufacturing facility in Bedford, Massachusetts to support “one of the largest advanced clinical gene therapy portfolios in the industry.”

Around 150 jobs will be created at the 110,000 square-foot facility, which is located close to Ultragenyx’s research and development operations in the Greater Boston area.

“In terms of capacity, our equipment is capable of producing adeno-associated virus (AAV) gene therapies at the 2,000-liter scale and can currently run up to 30 batches per year,” Dennis Huang, chief technical operations officer and executive vice president for gene therapy research and development at Ultragenyx told this publication.

The facility in Beford, MA. Image c/o Ultragenyx

“This facility will exclusively produce AAV gene therapy products to support our pipeline of investigational and commercial gene therapies for rare diseases,” he continued. “At Ultragenyx, we have developed one of the largest advanced clinical gene therapy portfolios in the industry leveraging our proprietary producer cell line (PCL) platform and expertise.”

The PCL platform aims to enable efficient and scalable production of AAV gene therapies through simpler workflows and lower material costs than traditional methods.

“We have engineered PCLs that stably produce high yields of viable, intact AAV vectors, while maximizing the production of full AAV capsids,” said Huang, adding the platform “is designed to support the safety of AAV therapy as it is applied to diseases that may require relatively higher doses – such as those impacting central nervous system (CNS) and muscle tissues.”

Ultragenyx has three gene therapies in Phase III: UX111 (formerly ABO-102) for Sanfilippo syndrome type A , DTX401 for the potential treatment of glycogen storage disease type Ia (GSDIa), and DTX301 being investigated for ornithine transcarbamylase (OTC) deficiency. Another gene therapy for Wilson disease is in Phase II.

“We designed this plant with the future in mind and we can double our current capacity if needed by activating a second manufacturing suite in the facility,” Huang said. While there are no plans to operate as a contract manufacturing organization, Ultragenyx is open to using the capacity for strategic partnerships if needed in the future.

The cost of the new facility has not been disclosed.