Amicus picks Brammer for preclinical, clinical gene therapy work

02-Jul-2019 - Last updated on 02-Jul-2019 at 14:06 GMT

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Rare disease drug developer to work with Brammer Bio – which Thermo Fisher acquired in March – on gene therapy programs against Batten disease.

Amicus Therapeutics, a company focused on rare metabolic diseases, announced a strategic manufacturing collaboration with Thermo Fisher Scientific and its recently acquired contract development and manufacturing organisation (CDMO) for gene therapies development, Brammer Bio.

Following the agreement, Brammer, which is now integrated into Thermo Fisher Pharma Services, will take over the clinical and commercial manufacturing of Amicus’ intrathecal AAV Batten disease gene therapy programs.

Specifically, the partners will work together on the preclinical and clinical-stage programs for CLN6, CLN3, and other potential Batten disease programs, which will be transferred to Thermo Fisher’s viral vector services business.

According to Amicus, the collaboration also includes the development of platform manufacturing capabilities for a ‘broader portfolio’ of adeno-associated virus (AAV) gene therapy programs.

John Crowley, CEO of Amicus said in a statement that the partnership is a ‘significant next step’ to deliver therapies for people with rare genetic diseases “as quickly as possible, especially in devastating diseases like Batten’s, where time is of the essence.”

Thermo Fisher’s establishment in the gene therapy space

Thermo Fisher acquired Brammer Bio in a $1.7bn deal signed in March 2019, aiming to expand its CDMO capabilities in the gene therapy market.

A spokesperson for Thermo Fisher told us at the time that the company “can meaningfully contribute to driving industry standardization in gene therapies to effectively support this rapidly growing market,” adding that around 60-70% of the market is outsourced to CDMOs.

In an interview during the BIO Convention in June 2019, the company’s president of commercial operation, Franco Negron, spoke about a new business model in the CDMO market, developing due to the rising demand of cell and gene therapies.