Ultragenyx opens gene therapy plant in Bedford
Facility will make treatments for rare diseases
Ultragenyx Pharmaceutical, the San Francisco Bay Area-based biopharmaceutical company, on Wednesday officially opened a 110,000-square-foot plant in Bedford to manufacture gene therapies, one of the most innovative but expensive approaches to treat serious inherited diseases.
Ultragenyx, whose pipeline of experimental gene therapies include treatments for Duchenne muscular dystrophy and Wilson disease — a rare inherited disorder that causes copper to accumulate in the liver, brain, and other vital organs — opened the plant on a 10.7-acre site off Route 3.
“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, executive vice president for gene therapy research and development at the Novato, Calif.-based firm.
The plant has about 120 fulltime employees and about 30 contractors, according to Sue Marrichi, vice president and site head. Ultragenyx has roughly 230 more employees in Woburn and Cambridge. It has a total of 1,350 worldwide in locations that include North America, Latin America, Europe, and Japan.
Marrichi declined to say how much the new plant cost. It employs workers with a wide range of educational backgrounds, from high school diplomas to PhDs and MBAs, she said. Several are graduates of Middlesex Community College, which has a campus in Bedford.
Since 2017, Ultragenyx has won approval of four drugs for ultrarare and rare diseases, including a metabolic disorder and a bone disorder. The 13-year-old firm has yet to win approval of a gene therapy.
The Food and Drug Administration has approved at least six gene therapies since 2017 that have transformed the outlook for diseases once considered hopeless. The therapies typically used viruses modified in a lab to deliver a functional gene to compensate for a defective one. They are also breathtakingly expensive.
Among the biotechs that have gotten gene therapies cleared recently is Somerville’s Bluebird Bio. In August, it won approval of Zynteglo, a $2.8 million one-time treatment for a rare inherited blood disease called beta thalassemia. A month later, it won approval of Skysona, a $3 million one-time treatment for the fatal degenerative brain disease cerebral adrenoleukodystrophy.
Another local biotech, Cambridge’s Sarepta Therapeutics, hopes to win approval this week of a gene therapy for Duchenne muscular dystrophy even though experts debate how effective it was in a clinical trial.
Although some companies might hire a contract manufacturing organization to make a gene therapy, Marrichi said Ultragenyx preferred to have its own plant to control the timing of production. “Otherwise, you’re at the mercy of the contract manufacturing facility,” she said.