Developing isogenic models of cardiovascular disease

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 rationalise many aspects of drug development, reducing the cost of bringing to market new personalized therapies.

Horizon recently launched an online support site, www.rAAVers.org, for scientists working with rAAV-mediated genome editing.