Horizon Discovery (Horizon), a provider of research tools to support the development of personalized medicines, has announced that it will commit resources to provide training and open access to its rAAV GENESIS human gene-editing platform for another 50 academic and not-for-profit research groups over the next five years, extending the GENESIS Gene Editing Consortium. This forms part of Horizon’s strategy to generate at least 2500 new X-MANTM (gene X- Mutant And Normal) models of cancer, neurodegenerative & cardiovascular disease. These models will support drug discovery researchers to understand how complex genetic diseases manifest themselves in real patients and help rationalize many aspects of drug development, and therefore the cost of bringing to market new personalized therapies.
The goal of the GENESIS Gene Editing Consortium is to advance the application of rAAV gene-editing in functional genomics and translational medicine. These 50 research groups will become members of the Consortium, joining rAAV GENESIS pioneers; the University of Washington, the University of Torino, Johns Hopkins University, the Translational Genomics Institute (TGen), the University of Minnesota, the University of Maryland, Case Western Reserve University and the University of Pittsburgh.
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.
Membership of the Consortium is free to all academic and not-for-profit organizations but requires a commitment to generate at least 10 new models per year and the entering into of a pre agreed technology access agreement. Interested organizations can apply anytime by writing to firstname.lastname@example.org.
Dr Chris Torrance, CSO and co-founder of Horizon, said “With the enormity of genetic variations that are known from global human genome sequencing efforts and the emerging need to decipher these variations for their role in predicting or progressing specific diseases; a similarly large-scale consortium approach is required to make sense of all this information. For this reason, Horizon aims to form a hub of expert academic researchers and institutes to create and study an un-heralded array of human disease models, and therefore accelerate the development of many more ‘targeted’ or ‘personalized’ medicines that could impact global unmet clinical needs such as cancer, neurodegeneration, cardiovascular and infectious diseases.”.
Dr Darrin M Disley, Executive Chairman of Horizon, said: “We will continue the open access approach we have historically taken to working with academia as we believe that this approach will rapidly generate the cellular disease models essential for the research of genetics-based diseases such as cancer. The Consortium provides a route for academic and not-for-profit organizations to both advance their fundamental research and generate license income via Horizon, providing a route to market for research materials and findings generated using our patented rAAV GENESIS platform.”