Leading researchers Steve Jackson and David Rubinsztein to apply rAAV gene editing technology in studies of oncology and neurodegenerative disease
Cambridge, UK, 11 March 2013: Horizon Discovery (Horizon), a leading provider of research tools to support the development of personalized medicines, today announced it has established two new Centers of Excellence (CoEs) for gene editing in Cambridge, UK. The new Centers are at the Gurdon Institute, Cambridge University with Prof. Steve Jackson FRS, and the Cambridge Institute for Medical Research with Prof. David Rubinsztein.
Prof. Jackson aims to establish Horizon’s rAAV genome editing technology (GENESIS™) as a core technology within his lab at the Gurdon Institute for generating isogenic cell models of cancer. They will focus on generating cell lines with specific mutations in genes involved in DNA repair and DNA damage signaling, to study their relationship to cancer progression.
Prof. Rubinsztein’s lab will apply GENESIS to generate isogenic cell line models for the study of autophagy (degradation of unnecessary or dysfunctional cellular components), which has been a focus since their discovery that autophagy regulates the levels a set of specific proteins that cause many neurodegenerative diseases.
“The increasing number of research groups of this caliber working with our genome editing technology is very exciting for Horizon,” said Dr Rob Howes, Principal Scientist, Horizon. “We are delighted to welcome both the Gurdon Institute and Cambridge Institute for Medical Research to our Centers of Excellence network and look forward to working with them on developing the cell lines.”
The new human isogenic cell lines generated by the CoEs will be exclusively licensed to Horizon in return for future product royalties. Horizon will also have an exclusive option to license new intellectual property developed. This forms part of Horizon’s strategy to generate at least 2,500 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.
These centers become part of the larger Horizon Centers of Excellence network, which includes high profile Institutions such as UCL, Yale University, the National Cancer Institute, Washington University in St Louis and the National Cancer Centre, Japan.
Horizon sponsors an online community portal, www.rAAVers.org, for scientists working with rAAV-mediated and other forms of 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 Company’s 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 500 X-MAN™ cell lines, the world’s first source of genetically-defined and patient-relevant human cell lines, accurately modeling the disease-causing mutations found in cancer patients. These ‘patients-in-a-test-tube’ 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 ‘right drugs’ for the ‘right patients’ 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.
GENESIS and X-MAN are registered trademarks of Horizon Discovery Limited.
Dr Rob Howes
Tel: +44 (0) 1223 655 584
Email: [email protected]
Tel: +44 (0) 7811 996 942
Email [email protected]