Horizon Discovery licenses CRISPR gene editing technology from the Broad Institute

Under the terms of the agreement, Horizon has rights to use the technology commercially for applications including development of research tools and reagents, and provision of services that utilize CRISPR, for example as part of Horizon’s GENASSIST™ offering. GENASSIST is a comprehensive product and service solution for CRISPR and rAAV gene editing, including guide RNA design and validation, donor design, and a wide range of off-the-shelf plasmids.

The acquisition of additional CRISPR IP is in line with the Company’s aims outlined at the time of its IPO and further strengthens Horizon’s position in the market for this cutting edge gene editing technology. Access to a broader CRISPR IP portfolio will allow the technology to be deployed extensively across Horizon’s products, services and leveraged R&D and enhance the attractiveness of the Horizon GENESIS™ offering to customers.

The Broad Institute patent represents the first granted patent1 for use of CRISPR technology in eukaryotic cells and is based on work by Dr. Feng Zhang in a paper published in Science in 20132. More recently Dr. Zhang has published compelling results3 indicating CRISPR/Cas9-mediated gene editing can be employed in high-throughput genome-wide screens. This technology has the potential to revolutionize functional genomics and will also be offered as a service by Horizon.

“Horizon is committed to gaining access to the widest range of CRISPR IP possible.  We have taken this approach to ensure that our customers and partners are secure in the knowledge that they have the freedom to pursue their research and commercial goals when they choose to work with us. Building upon the Charpentier IP, in-licensed in early May, the Broad patent represents another important part of this program, and we are very pleased to be able to announce its addition to our growing portfolio of CRISPR licenses,” commented Dr. Darrin Disley, CEO, Horizon Discovery Group plc.

“Cas9 is revolutionizing many areas of biological research,” said Dr. Zhang, a core member of the Broad Institute and a pioneer of the CRISPR technology. “This technology has a lot of potential and we are excited to work with partners like Horizon to help put these powerful genome editing tools into the hands of researchers.”

In line with its strategy outlined at the time of its IPO, Horizon continues to review the gene editing field for further product expansion and IP licensing opportunities.


1. US Patent #8,697,359

2. Science, 2013 Feb 15; 339(6121):819-23

3. Science, 2014 Jan 3; 343(6166):84-7



About Horizon Discovery Group plc

Horizon (LSE:HZD) is a revenue-generating life science company supplying research tools to organizations engaged in genomics research and the development of personalized medicines. Horizon has a diverse and international customer base approaching 800 organizations, including major pharmaceutical, biotechnology and diagnostic companies as well as leading academic research centres. The Company supplies its products and services into multiple markets, estimated to total in excess of £29 billion by 2015.

Horizon’s core capabilities are built around its proprietary translational genomics platform, GENESIS™, a high-precision and flexible suite of gene editing tools able to alter almost any endogenous gene sequence of human or mammalian cell-lines. Horizon has applied GENESIS™ to create over 550 X-MAN™ cell lines, accurately modeling the disease-causing mutations found in genetically based diseases. These ‘patients-in-a-test-tube’ are being used by customers to identify the effect of individual or compound genetic mutations on drug activity, patient responsiveness, and resistance, which may lead to the successful prediction of which patient sub-groups will respond to currently-available and future drug treatments.

In addition, Horizon provides GENASSIST™ CRISPR and rAAV gene editing tools, custom cell line generation services for research and bioproduction applications, quantitative molecular reference standards, contract research and custom screening services, haploid cell lines (through Horizon’s partner Haplogen), and custom shRNA development services and off-the-shelf validated shRNA (through Horizon’s partner Sirion).

Horizon is headquartered in Cambridge, UK and is listed on the London Stock Exchange’s AIM market under the ticker “HZD”, for further information please visit: www.horizondiscovery.com
 
About the Broad Institute of MIT and Harvard
The Eli and Edythe L. Broad Institute of MIT and Harvard was launched in 2004 to empower this generation of creative scientists to transform medicine. The Broad Institute seeks to describe all the molecular components of life and their connections; discover the molecular basis of major human diseases; develop effective new approaches to diagnostics and therapeutics; and disseminate discoveries, tools, methods, and data openly to the entire scientific community.

Founded by MIT, Harvard, and its affiliated hospitals, and the visionary Los Angeles philanthropists Eli and Edythe L. Broad, the Broad Institute includes faculty, professional staff and students from throughout the MIT and Harvard biomedical research communities and beyond, with collaborations spanning over a hundred private and public institutions in more than 40 countries worldwide. For further information about the Broad Institute, go to http://www.broadinstitute.org.

About the engineered CRISPR-Cas9 system
CRISPRs (Clustered Regularly Interspaced Short Palindromic Repeats) have recently been harnessed as genome editing tools in a wide range of species. The engineered CRISPR-Cas9 system allows researchers to mutate or change the expression of genes in living cells, including those of humans. The family of Cas9 nucleases (also known as Cas5, Csn1, or Csx12) recognizes DNA targets in complex with RNA guides. Researchers can now harness the engineered system to home in on specific nucleic acid sequences and cut the DNA at those precise targets. The cuts modify the activity of the targeted genes, allowing researchers to study the genes' function.

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