Horizon Discovery licenses AAV gene delivery technology from Stanford University


03-07-2014

Horizon Discovery™ Group plc, a leading provider of research tools to support translational genomics and the development of personalized medicines, today announced it has entered into a non-exclusive license agreement with Stanford University to access intellectual property related to the use of a proprietary serotype of AAV (Adeno-Associated Virus) for commercial applications involving homologous recombination in gene editing.

Created by Professor Mark Kay at Stanford this artificial serotype of AAV known as AAV-DJ effectively transduces (infects) a broader range of mammalian cells (>80%) than any naturally occurring AAV serotype, enabling Horizon to target an increased range of cell types. Combined with Horizon’s existing exclusive license for use of rAAV (recombinant Adeno-Associated Virus) from the University of Washington, the Company is the sole source of this effective and highly precise mechanism for gene editing.

“Horizon is committed to ensuring our customers are supported by the widest range of gene editing tools secured by appropriate licenses, enabling us to offer the most complete suite of gene editing products and services to meet their needs,” commented Eric Rhodes, CTO, Horizon.

In February 2014 Horizon announced the launch of its GENASSIST™ range of gene editing kits and reagents that enable easier, robust implementation of CRISPR and rAAV gene editing experiments. The current GENASSIST offering comprises both off-the-shelf reagents for using CRISPR editing technology and a unique kit combination of these reagents to allow customers to generate their own CRISPR-ready cell lines that constitutively express Cas9-nickase.

Horizon offers researchers an unrivalled toolbox capable of performing rapid functional genomics experiments, and creating high-precision human disease models for deployment at all stages of drug discovery and diagnostic development.  Horizon employs three genome editing technologies (AAV, ZFN and CRISPR) for custom client-led projects, and is continually expanding upon its own menu of over 550 genetically-defined off-the-shelf disease model cell lines and related products.

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



About Horizon Discovery

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).

In June 2014 Horizon launched a scheme under which academic researchers can access GENASSIST for free, as part of a CRISPR knockout generation programme. The scheme is open to academic researchers for human genes, and includes free guide design with gUIDEbook™, and free cloning into an all-in-one vector expressing Cas9. The aim of the scheme is to promote the use of the ground breaking CRISPR technology and to expand the portfolio of knock out cell lines available to the scientific community as disease models and compound screening tools.

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   

 
 *******

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

Zyme Communications (Trade and Regional Media)
Katie Odgaard
Tel: +44 (0)7787 502 947
Email: katie.odgaard@zymecommunications.com

Consilium Strategic Communications (Financial Media and Investor Relations)
Amber Bielecka / Mary-Jane Elliott / Jessica Hodgson / Matthew Neal
Tel: +44 (0) 20 3709 5701
Email: horizon@consilium-comms.com

Panmure Gordon (UK) Limited (NOMAD)
Corporate Finance: Freddy Crossley / Duncan Monteith / Fred Walsh
Broking: Tom Salvesen
Tel: +44 20 7886 2500
_____________________________________________________________

 

Horizon Discovery Group plc, a UK headquartered global life science company, is a world leader in gene editing and gene modulation.

Horizon Discovery