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20 April, 2011 - 10:47 By Staff Reporter

Cambridge-US link on personalised medicines


Horizon Discovery, the Cambridge UK personalised medicines pioneer, has agreed a three-year collaboration with the Fox Chase Cancer Center in Philadelphia, that will create a new centre of excellence in mammalian gene editing.

The focus of the collaboration is to apply rAAV gene targeting in mouse embryonic stem (ES) cells and discover 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 alliance. 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 rationalise 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 centre 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.

Last December, Horizon announced that it planned 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. Fox Chase Cancer Center is one of the leading cancer research and treatment hubs in the US. 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. Its researchers have won the highest awards in their fields, including two Nobel Prizes.

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