Horizon’s CoE program encompasses academic and not-for-profit research groups or laboratories to which Horizon commits resources to provide training and open access to its proprietary rAAV-mediated human gene-editing platform, GENESIS™. The University of Leicester is the first of Horizon’s Centers of Excellence to focus on cardiovascular disease.
The new human isogenic cell lines generated by the University of Leicester 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 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.
“Horizon’s rAAV-mediated genome editing can fundamentally alter the genome of cells, leading to the creation of patient-relevant disease models. The use of these models in oncology is well established, and has predicted patient responses to targeted therapies both during drug development and in the clinic,” commented Dr Rob Howes, Principal Scientist, Horizon Discovery. “By developing the application of this technology to other disease areas such as cardiovascular disease, we aim to lead the development of personalized medicines in these areas.”
Prof Nilesh Samani, British Heart Foundation Professor of Cardiology and Head of Department of Cardiovascular Sciences at the University of Leicester, commented: “The opportunity to apply Horizon’s GENESIS technology for development of cardiovascular disease cell lines offers an exciting progression in the study of the disease. We look forward to working with Horizon on this program.”
Dr Tom Webb, British Heart Foundation Lecturer in Cardiovascular Genomics, at the University of Leicester, said: “We are delighted to have this partnership with Horizon. By using rAAV-mediated genome editing we can create cell models to investigate how specific genetic variants contribute to the development of cardiovascular disease.”
The Centers of Excellence are part of 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.
Contacts:
At Horizon:
Dr Rob Howes
Principal Scientist
Tel: +44 (0) 1223 655 584
Email: r.howes@horizondiscovery.com
Media enquiries:
Katie Odgaard
Zyme Communications
Tel: +44 (0)7787 502 947
Email katie.odgaard@zymecommunications.com
At the University of Leicester:
Nilesh Samani
British Heart Foundation Professor of Cardiology
Head of Department of Cardiovascular Sciences, University of Leicester
Director Leicester NIHR Biomedical Research Unit in Cardiovascular Disease
Email: njs@le.ac.uk
Tom Webb
British Heart Foundation Lecturer in Cardiovascular Genomics
Department of Cardiovascular Sciences, University of Leicester
Tel: +44 (0)116 258 3716
Email: tw126@le.ac.uk
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 400 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.