CRISPR Collaboration with Fulcrum Therapeutics

Horizon Discovery 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."

ENDS

For further information from Horizon Discovery Group plc, please contact:

Consilium Strategic Communications (Financial Media and Investor Relations)

Mary-Jane Elliott / Susan Stuart / Matthew Neal / Melissa Gardiner

Tel: +44 (0) 20 3709 5701

Email: [email protected]

Zyme Communications (Trade and Regional Media)

Katie Odgaard

Tel: +44 (0)7787 502 947

Email: [email protected] 

Numis Securities Limited (Joint Broker and NOMAD)

Michael Meade / Freddie Barnfield

Tel: +44 (0) 207 260 1000

RBC Capital Markets (Joint Broker)

Paul Tomasic / Marcus Jackson

Tel: +44 (0) 20 7653 4000

For further information from Fulcrum Therapeutics Inc., please contact:

Dan Quinn, Ten Bridge Communications (Trade and Regional Media)

Tel: (1) 781-475-7974

Email: [email protected]

About Horizon Discovery Group plc www.horizondiscovery.com

Horizon Discovery Group plc (LSE: HZD) ("Horizon"), is a world-leading gene editing company that designs and engineers genetically-modified cells and then applies them in research and clinical applications that advance human health.

Horizon's core capabilities are built around its proprietary translational genomics platform, a highly precise and flexible suite of gene editing tools (rAAV, ZFN and CRISPR) able to alter almost any gene sequence in human or mammalian cell-lines.

Horizon offers over 23,000 catalogue products and related research services, almost all of which are based on the generation and application of cell and animal models that accurately recapitulate the disease-causing genetic anomalies found in diseases like cancer. Horizon's commercial offering has been adopted by c. 1,400 unique research organizations in over 50 countries as well as in the Company's own R&D pipeline to support a greater understanding of the genetic drivers of disease and the development of molecular, cell and gene therapies that can be prescribed on a personalised basis.

Horizon is headquartered in Cambridge, UK, and is listed on the London Stock Exchange's AIM market under the ticker "HZD".

About Fulcrum Therapeutics www.fulcrumtx.com

Fulcrum Therapeutics is a biotechnology company developing new medicines to deliver a new future to patients and their families by transforming gene regulation in disease. Fulcrum's therapies are based on 3 modulating gene regulation via control of genetic on and off switches of disease genes. Fulcrum was launched in 2016 by Third Rock Ventures, and is headquartered in Cambridge, Mass.