Thermo Fisher and Catalent to manufacture gene therapies being developed by Amicus

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 CDMOs.  

The New Jersey-based biotech on Monday announced it had 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. It followed that up on Tuesday with an announcement that it had struck a similar deal with Paragon Gene Therapy, a unit of Catalent Biologics. 

With Thermo Fisher it is working on its Batten disease treatment programs while Catalent will be focused on Amicus'  adeno-associated virus (AAV) manufacturing platform and its preclinical gene therapy program for Pompe disease. 

"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 teams and strategic partners in the tech transfer process. 

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 to buy 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 acquired 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 and Catalent are new to gene therapies as well. Thermo Fisher 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. 

Weeks later, Catalent made its move into gene therapy manufacturing with a $1.2 billion deal for Paragon Bioservices. The all-cash deal came with a newly completed commercial-scale manufacturing facility near Baltimore and its 380 employees.