Horizon Discovery, Fulcrum partner for novel CRISPR-based target discovery in genetic diseases

Fulcrum will use Horizon's CRISPR screening platform to find targets for regulating gene expression in diseases with no current treatment options.

The initial program features two projects with the potential for additional future projects.

The work will be carried out under a full-time equivalent and consumables model in a program expected to run between five and seven months.

Horizon has been selected as Fulcrum’s preferred CRISPR-based screening platform service provider. There is potential for additional screens to identify targets for further rare diseases after the initial program.

Financial and other terms of the collaboration were not disclosed.

Horizon claims that its CRISPR-based screening platform, bioinformatics, and cell line libraries offer a novel way to evaluate the regulation of genes and their role in disease. 

Horizon Discovery CEO Darrin Disley 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.

"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."

Fulcrum Therapeutics was launched in July 2016 by Third Rock Ventures to discover and develop small molecules that modulate the on/off control mechanisms that regulate genes.

The company’s therapies are based on modulating gene regulation through control of genetic on and off switches of disease genes.

Image: Fulcrum will use Horizon's CRISPR screening platform to find targets for regulating gene expression in diseases with no current treatment options. Photo: courtesy of jk1991 at FreeDigitalPhotos.net.