Horizon and fledgling Fulcrum team up on using CRISPR for genetic diseases

Just a few months after launching with a $55 million Series A round, Third Rock upstart Fulcrum Therapeutics has signed its first major partnership with the U.K.’s Horizon Discovery as they look to put CRISPR to use for rare, genetic diseases.

Details were a little thin on the ground, but the two said in a statement that the initial program “consists of two projects with the potential for additional future projects.” Financial details were not revealed. 

Horizon Discovery Group (LSE: HZD), which is aiming to become a big player when it comes to the application of gene editing technologies, said it will use its CRISPR-based screening platform to identify new targets for regulating gene expression. The program will initially focus on genetic diseases where no effective treatment options currently exist.

Fulcrum, led by former Epizyme ($EPZM) CEO and Merck ($MRK) veteran Robert Gould, is currently engaged in early-stage work on two monogenic diseases: Fragile X syndrome (FXS) and a form of muscular dystrophy called facioscapulohumeral muscular dystrophy (FSHD).

The biotech is looking to use pluripotent stem cells, derived from skin, to mimic the cellular processes that have gone wrong in these diseases with altered gene expression. It is working on using small molecules to modulate this in its first two targets.

The company recently told FierceBiotech that it uses screening tools, such as CRISPR-Cas9 and chemical probe libraries, to dissect gene regulatory mechanisms in cellular models of diseases.

Under the collab, Horizon said it will apply its CRISPR platform and know-how to identify gene regulation targets for Fulcrum, which will then go on to try to use these for the development of next-gen therapies.

The work between the two is expected to run between 5 and 7 months, although Fulcrum says it has chosen Horizon as its preferred CRISPR-based screening platform service provider, and there is “potential for further screens to identify targets for additional rare diseases after this initial programme.”

Dr. Darrin Disley, CEO of Horizon Discovery, said: “CRISPR-based screening promises to be a powerful tool for the identification of novel targets and the collaboration with Fulcrum demonstrates the potential for this technology to be used in areas outside of oncology.” He continued: “This partnership confirms Horizon’s role as a preferred partner not only for established biotechnology and pharmaceutical companies, but also for start-ups looking for long-term collaborations rather than having to build in-house capabilities.”

Robert Gould, president and CEO of Fulcrum Therapeutics, added: “Horizon’s reputation in gene editing and its applications, including CRISPR-based screening, is unparalleled. It was natural for Fulcrum to turn to Horizon as a long-term partner. With their deep scientific expertise, and with a broad IP portfolio underpinning their work, we are confident in a productive and exciting collaboration.”