Horizon Discovery Ltd. And Fulcrum Therapeutics Form Collaboration For Novel CRISPR-Based Target Discovery In Genetic Diseases

•Horizon will deploy its CRISPR-based screening platform to identify novel targets for rare genetic diseases

•The collaboration confirms Horizon’s role as a preferred partner not only for established biotechnology and pharmaceutical companies, but also for start-ups

•The initial programme consists of two projects with the potential for additional future projects

Cambridge, UK, 6 September 2016: Horizon Discovery Group plc (LSE: HZD) ("Horizon"), the world leader in the application of gene editing technologies, today announced a new collaboration with Fulcrum Therapeutics Inc., (“Fulcrum”), a US-based firm founded in July 2016 by Third Rock Ventures. The collaboration will utilize Horizon’s world-leading CRISPR-based screening platform to identify novel targets for regulating gene expression. The programme will initially focus on genetic diseases where no effective treatment options currently exist.

Horizon’s CRISPR-based screening platform, sophisticated bioinformatics, and cell line libraries provide a novel and highly efficient way to examine the regulation of genes and their role in disease. In this collaboration, Horizon will apply its platform and extensive know-how to identify novel gene regulation targets for further exploration by Fulcrum for the development of next-generation therapies.

The work will be engaged under a Full-time Equivalent (FTE) plus consumables model in a programme expected to run between five and seven months. Fulcrum 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, Chief Executive Officer, Horizon Discovery commented, “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, Ph.D. President and Chief Executive Officer, Fulcrum Therapeutics, commented, “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.”

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