Ultragenyx is focused on developing and commercializing novel therapies for rare and ultrarare diseases. It said it has a “large and diverse clinical pipeline of late-stage gene therapies for treating rare genetic conditions.”

“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,” said Dennis Huang, chief technical operations officer and executive vice president for gene therapy R&D.

Ultragenyx plans to hire 120 new employees at the facility, adding to more than 350 people employed across its Massachusetts sites in Bedford, Woburn and Cambridge.

Japan’s Daiichi Sankyo has selected Ultragenyx’ proprietary Adeno-associated virus (AAV)-based gene therapy manufacturing technologies, including its Pinnacle producer cell line platform, for its internal gene therapy programs.

US life science company Solid Biosciences is also working with Ultragenyx to advance new AAV-based gene therapies for Duchenne muscular dystrophy.

Author: Elaine Burridge, Freelance Journalist