Cambridge, UK, 19th April 2011: Horizon Discovery (Horizon), a leading provider of research tools to support the development of personalized medicines, has announced that it has entered into a three-year collaboration with the Fox Chase Cancer Center (Fox Chase).
The focus of the collaboration is to apply rAAV gene targeting in mouse embryonic stem (ES) cells, and for the discovery of genes involved in resistance to EGFR-targeted therapies in human cancer, such as PI3K.
The collaboration will generate new human isogenic disease models and mouse knock-out models that will be exclusively licensed by Fox Chase to Horizon in return for future product royalties. Horizon will also have an exclusive option to license new intellectual property developed during the term of the collaboration. This forms part of Horizon’s strategy to generate at least 2500 new X-MANTM (gene X- Mutant And Normal) models of cancer, neurodegenerative, and cardiovascular disease. These models will support drug discovery researchers in their effort 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.
As part of the collaboration, Horizon and Fox Chase will establish a Center of Excellence in gene editing that will advance the application of rAAV gene-editing in functional genomics and translational medicine within in vivo mouse models, further extending the range of applications and models within the GENESIS Gene Editing Consortium. In December 2010, Horizon announced that it plans to commit resources to provide training and open access to its proprietary gene-editing platform to another 50 academic and not-for-profit research groups over the next five years.
At the heart of the GENESIS platform is the use of rAAV vectors that have a unique and powerful property in performing accurate and efficient gene-editing functions in human cells by switching on a natural and high-fidelity DNA-repair mechanism called homologous recombination (HR). When harnessed using rAAV gene-editing vectors, HR allows the precise alteration of any DNA sequence, permitting the correction of genetic defects in gene therapy applications, or the accurate modeling of genetic diseases in human cells in vitro.
Dr Rob Howes, Principal Scientist and Center of Excellence Program Manager at Horizon, said: “EGFR-targeted therapies, such as Erbitux and Vectibix, have been very effective in treating human cancers but there is a set of patients which are resistant to these treatments. Our collaboration with Fox Chase will identify genes which are involved in this resistance mechanism and provide novel targets for cancer treatment. The ability of rAAV to edit the genome of human cells is now routine. Establishing the rAAV Center of Excellence at Fox Chase will enable us to extend the applicability of rAAV to mouse gene targeting.”
Dr Jonathan Chernoff, Chief Scientific Officer at Fox Chase, commented: “These efforts will allow us to more realistically model how combining new signaling inhibitors affects certain drug-resistant cancers. In addition, if we are able to use rAAV technology to modify mouse ES cells, this could open the door to rapid, high throughput gene editing in the mouse.
About Fox Chase Cancer Center (www.foxchase.org)
Fox Chase Cancer Center is one of the leading cancer research and treatment centers in the United States. Founded in 1904 in Philadelphia as one of the nation’s first cancer hospitals, Fox Chase was also among the first institutions to be designated a National Cancer Institute Comprehensive Cancer Center in 1974. Fox Chase researchers have won the highest awards in their fields, including two Nobel Prizes. Fox Chase physicians are also routinely recognized in national rankings, and the Center’s nursing program has received the Magnet status for excellence three consecutive times. Today, Fox Chase conducts a broad array of nationally competitive basic, translational, and clinical research, with special programs in cancer prevention, detection, survivorship, and community outreach. For more information, visit Fox Chase’s Web site at www.foxchase.org or call 1-888-FOX CHASE or (1-888-369-2427).
About Horizon Discovery http://www.horizondiscovery.com
Horizon Discovery is a leading provider of research tools to support the development of personalized medicines. The Company’s proprietary virally-mediated gene-engineering technology, GENESIS™, enables any endogenous gene in a human cell-line to be altered quickly, reliably and without introducing unwanted and confounding genotypes and/or phenotypes. Using GENESIS, Horizon Discovery has developed X-MAN™ (gene-X; Mutant And Normal) cell models, which are the world’s first source of genetically-defined and patient-relevant human cell lines.
The Company’s broad panel of over 300 different X-MAN cell lines, which have been referred to as “patients-in-a-test-tube,” accurately model the disease-causing mutations in cancer patients. The X-MAN cell lines are being used by academic and industry leaders to identify the effect of individual mutations on drug activity, patient responsiveness and resistance, and to successfully predict which patient sub-groups will respond to currently-available and future drug treatments. This supports the design of shorter, more focused clinical trials, making it possible to identify the ‘right drugs’ for the ‘right patients’ based upon the unique genetic mutations that define their disease.
Founded in 2007 by Dr Chris Torrance and Professor Alberto Bardelli, and with headquarters in Cambridge, UK, the company builds on a long heritage in gene-engineering, cancer research and translational medicine, and draws on intellectual property and know-how from the University of Washington, the Johns Hopkins University, the University of Maryland, the University of Minnesota and the Department of Oncological Sciences at the University of Torino Medical School. Over 150 scientific papers relating to the use of X-MAN cell line pairs generated using GENESIS have been published, and the technology has won numerous awards including; Medical Futures Innovation Award for Cancer (2008), UK iaward of the Year (2009), and East of England Killer Technology (2010).
For more information please contact:
At Horizon:
Dr Darrin M Disley
Executive Chairman, Horizon Discovery
+44 (0)1223 303049 or +44 (0)7786 133893
d.disley@horizondiscovery.com
Media enquiries for Horizon:
Lorna Cuddon
Zyme Communications Ltd
+44 (0)7811 996 942
lorna.cuddon@zymecommunications.com
