Horizon Discovery licenses CRISPR/Cas9 gene editing technology from ERS Genomics

Under the terms of the agreement, Horizon has rights to use the technology, which is based on the work of Dr. Emmanuelle Charpentier and her colleagues, for research applications including development and sale of research tools, kits and reagents, performance of research services, creation of genetically modified disease model cell lines, development and production of reference standard material for molecular diagnostics and for internal target identification and validation research efforts.

The acquisition of this important CRISPR IP is in line with the Company’s aims outlined at the time of its IPO and further strengthens Horizon’s long-term 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 further enhance the attractiveness of the Horizon GENESIS™ offering to customers.

“Horizon Discovery’s ambition is to be the market leader in CRISPR technology, and by expanding our portfolio of intellectual property rights in this area we aim to ensure that our customers, both now and in the future, will have unencumbered access to this innovative new gene editing technology,” said Dr. Darrin Disley, Chief Executive Officer of Horizon Discovery Group. “We believe that the ERS Genomics IP, based on the work of Dr. Emmanuelle Charpentier and her colleagues, is particularly important and so we are very pleased to be able to add it to our expanding portfolio of patent rights in this area on behalf of our customers.”

Professor Emmanuelle Charpentier was principal co-author on a seminal publication[1] suggesting how the CRISPR/Cas9 system could be adapted from use in bacteria for broad application in mammalian genome editing. She is currently serving as head of the Department of Regulation of Infectious Disease at the Helmholtz Centre for Infection Research.

Horizon continues to review the gene editing field for further product expansion and IP licensing opportunities.

Reference [1] Science. 2012 Aug 17; 337(6096):816-21.

 

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 centers. 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 CRISPR

“CRISPR”, (clustered regularly interspaced short palindromic repeats) is a new RNA-guided gene-editing platform that makes use of a bacterially derived protein (“Cas9”) and a synthetic guide RNA (“gRNA”) to introduce a break at a specific location within the genome. While bacterial Cas9 is capable of introducing double strand breaks similar to Zinc Finger Nucleases, a modified version introduces only single strand “nicks” to the DNA, which can be exploited to introduce subtle genetic changes while avoiding off-target effects common to double strand breaks. Editing is achieved by transfecting a cell with the Cas9 protein along with a specially designed gRNA that directs the cut through hybridization with its matching genomic sequence.

The use of CRISPR has been described in several peer-reviewed publications in recent years. Horizon Discovery is deploying the platform alone and in combination with rAAV in its contract research services and cell line products business units.

 
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Contacts:

Horizon Discovery Group plc
Dr Darrin M Disley, Chief Executive Officer
Richard Vellacott, Chief Financial Officer and Senior Vice President, Operations
Tel: +44 (0) 1223 655 580


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Katie Odgaard
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Tel: +44 (0)7787 502 947

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Amber Bielecka / Mary-Jane Elliott / Jessica Hodgson / Matthew Neal
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