Under the agreement, Thermo Fisher will provide Amicus with immediate clinical and commercial manufacturing capabilities and capacity for Amicus’ intrathecal AAV Batten disease gene therapy programs.
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Amicus Therapeutics signed a strategic manufacturing deal with Thermo Fisher Scientific. Specifically, Amicus established a manufacturing collaboration with Thermo Fisher’s Brammer Bio, which Thermo Fisher acquired in March 2019.
Under the agreement, Thermo Fisher will provide Amicus with immediate clinical and commercial manufacturing capabilities and capacity for Amicus’ intrathecal AAV Batten disease gene therapy programs. In addition to the Thermo Fisher deal, Amicus has developed a plasmid supply network as well as various long-term supply deals from industry leading plasmid suppliers.
The deal with Thermo Fisher has three components. The first is the Batten disease programs. The R&D production technology and capabilities related to CLN6, CLN3 and other possible Batten disease programs will be transferred to and developed by Thermo Fisher’s viral vector business. The second is the CLN6 Batten disease gene therapy, whose existing process will be moved to Thermo Fisher and be modified for a GMP process for clinical and commercial-scale manufacturing. And the two companies plan to collaborate to develop platform manufacturing abilities for a broader portfolio of AAV gene therapy.
“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 disease like Batten’s, where time is of the essence,” stated John F. Crowley, chairman and chief executive officer of Amicus. “With our decades of experience in biologics manufacturing, we understand the importance and complexities of technology transfer during the drug development process. We look forward to embedding our Amicus team and other strategic partners in the tech transfer process to Thermo Fisher, and to further augmenting our manufacturing capabilities and partnerships across our entire gene therapy portfolio.”
Batten disease is also called Neuronal Ceroid Lipofuscinosis (NCL), a family of rare diseases caused by autosomal recessive mutations. These mutations prevent the body’s cells from properly disposing of waste. There are 14 known types of Batten disease. Symptoms include progressive neurological impairment, visual impairment and seizures.
Thermo Fisher Scientific acquired Brammer Bio in March for $1.7 billion. Brammer Bio is a contract development and manufacturing organization (CDMO) focusing on manufacturing viral vectors for gene and cell therapies.
At its locations in Massachusetts and Florida, Brammer has about 600 staffers. At the time of the acquisition, Marc N. Casper, president and chief executive officer of Thermo Fisher, stated, “Gene therapy is an area of increasing focus for our customers and is fast-evolving given its potential to treat a range of genetic disorders. The combination of Brammer Bio’s viral vector capabilities with our GMP production expertise and proprietary bioprocessing and cell culture technologies uniquely positions us to partner with our customers to drive the evolution of this incredibly fast-growing market.”
No financial terms of the Amicus and Thermo Fisher deal were released.
“We see this as a strong strategic relationship to enable manufacturing of a deep gene therapy pipeline across various stages of development for people with devastating genetic diseases,” stated Chris Murphy, Thermo Fisher’s vice president and general manager, viral sector services. “These therapies have the potential to transform patients’ lives and, as an organization, we are delighted to support partners such as Amicus as they advance tomorrow’s potential medicines.”
