Horizon Discovery and UCL Institute of Neurology Collaborate in rAAV-mediated Genome Editing

Program will focus on developing human isogenic cell lines modeling Huntington¹s disease

Cambridge and London, UK, 08 November 2012: Horizon Discovery (Horizon), a leading provider of research tools to support the development of personalized medicines, today announced it has entered into a research collaboration for gene editing at the Institute of Neurology, University College London (UCL). The program of research will focus on the creation of human isogenic disease model cell lines with insertion of Huntington¹s disease-causing triplet repeats.

The UCL collaboration contributes to an academic not-for-profit research group to which Horizon commits resources to provide training and access to its proprietary rAAV-mediated human gene-editing platform, GENESIS . The program at UCL represents a new disease area for Horizon, and the insertion of triplet repeats into a wild type genome is a novel genetic alteration application for the GENESIS technology.

Prof. Sarah Tabrizi, Professor of Clinical Neurology at the UCL Institute of Neurology, global Principal Investigator of the TRACK-HD study and leader of all UCL Huntington¹s disease projects, said: ³Although mouse models have given important insights, it is important when studying human disease to use a broad approach, including cultured cells. The ability to accurately introduce the triplet repeat mutation into human somatic cell lines offers exciting possibilities in the study of Huntington¹s disease and potential therapies.²

³The use of patient-relevant disease models created by Horizon¹s rAAV-mediated genome editing technology is well established in oncology,² commented Dr Rob Howes, Principal Scientist, Horizon Discovery. ³We believe that by developing the application of this technology to other disease areas such as Huntington¹s disease, we can provide a vital tool for understanding, preventing and treating those diseases.²

The new human isogenic cell lines generated by UCL will be exclusively licensed to Horizon.  Horizon will also have an exclusive option to license new intellectual property developed. This forms part of Horizon¹s strategy to generate at least 2500 new X-MAN  (gene X- Mutant And Normal) models of cancer, neurodegenerative, and cardiovascular disease. These models support drug discovery researchers in their efforts to understand how complex genetic diseases manifest themselves in real patients, and help rationalize many aspects of drug development, reducing the cost of bringing to market new personalized therapies.

The UCL collaboration will complement the GENESIS Gene Editing Consortium, which includes rAAV GENESIS pioneers the National Cancer Institute, Cambridge University, Yale University, and Dana-Farber Cancer Institute.

Horizon recently launched an online support site, www.rAAVers.org, for scientists working with rAAV-mediated genome editing.

About Horizon Discovery http://www.horizondiscovery.com/

Horizon Discovery Limited (Horizon) is a leading provider of research tools to support translational genomics research and the development of personalized medicines. The Company¹s proprietary rAAV gene-editing technology, GENESIS , is industry leading. Using GENESIS, Horizon is able to alter any endogenous gene sequence of a human or mammalian cell-line quickly, reliably and without introducing unwanted and confounding genotypes and/or phenotypes.

Horizon has applied GENESIS to create over 400 X-MAN  cell lines, the world¹s first source of genetically-defined and patient-relevant human cell lines, accurately modeling the disease-causing mutations found in cancer patients. These Œpatients-in-a-test-tube¹ are being used by academic and industry leaders to identify the effect of individual or compound genetic mutations on drug activity, patient responsiveness, and resistance, leading to the successful prediction of which patient sub-groups will respond to currently-available and future drug treatments. This enables the design of shorter, more focused, and less expensive clinical trials, ultimately providing the tools to identify the Œright drugs¹ for the Œright patients¹ based upon the unique genetic mutations that define their disease.
In addition to the X-MAN cell lines, Horizon provides GENESIS and X-MAN  derived products and services, with industrial application in: bio-pharmaceutical process optimization; clinical diagnostic development; drug discovery & development; and the provision of reference standards for genomic-based clinical research platforms.

At Horizon:
Dr Rob Howes
Principal Scientist
Tel: +44 (0) 1223 655 584
Email: [email protected]

Media enquiries for Horizon:
Katie Odgaard
Zyme Communications
Tel: +44 (0)7787 502 947
Email [email protected]

Media enquiries for UCL:
David Weston
Media Relations Manager
Tel: +44 (0) 20 3108 3844
Email: [email protected]