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Horizon and University of Leicester Form CoE in rAAV-mediated Genome Editing

Published: Monday, July 16, 2012
Last Updated: Monday, July 16, 2012
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Collaboration will focus on developing isogenic models of cardiovascular disease.

Horizon Discovery (Horizon) has announced that it has established a Center of Excellence (CoE) for gene editing at the University of Leicester, UK.

The program will focus on the creation of isogenic cell lines that contain ‘risk’ or ‘protective’ SNPs associated with cardiovascular disease, identified from GWAS studies.

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.

Dr Howes continued, “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.”


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