Amicus Therapeutics has already forked over $ 100 million for 10 gene therapies. It is building a new R&D operation to advance them. But when it comes to manufacturing, it is turning to a CDMO.
The New Jersey-based biotech today announced it has an arrangement with Thermo Fisher Scientific’s gene therapy unit to provide clinical supply and commercial production of its gene therapies to cure intrathecal AAV Batten diseases, as it works to get them to the market.
“As we advance one of the industry’s leading gene therapy pipelines, our partnership with Brammer Bio, now part of Thermo Fisher, is a significant next step in fulfilling our manufacturing strategy so that we can deliver novel gene therapies to more people living with rare genetic diseases as quickly as possible, especially in devastating diseases like Batten’s, where time is of the essence,” Amicus CEO John F. Crowley, said in a statement.
He said the company will “embed” its team and strategic partners in the tech transfer process to Thermo Fisher.
New Jersey’s Amicus has been working on drugs to stabilize lysosomal storage disorders (LSDs) like Fabry disease and Pompe disease for a few years. Last year it nabbed FDA approval last for Galafold to treat Fabry disease. But with the $ 100 million deal with Celenex, it has a chance to cure the rare conditions.
Celenex has been working on gene therapies for lysosomal disorder Batten disease. Among the programs Amicus bought are gene therapies that target the CLN6 and CLN3 forms of Batten.
Earlier this year, Amicus said it will build a 75,000-square-foot facility in Philadelphia to serve as the global headquarters for Amicus’ scientific work and the home of its gene therapy leadership team.
Thermo Fisher is new to gene therapies as well. The company earlier this year ponied up $ 1.7 billion to nab viral vector producer Brammer Bio, giving it entry into the field. It acquired manufacturing locations in Cambridge, Massachusetts and Florida, along with 600 employees in the deal.