Institutions will generate human isogenic diseases models.

Horizon Discovery is making its rAAV gene-editing technology, Genesis™ available to the National Cancer Institute (NCI) and National Human Genome Research Institute (NHGRI), for use in generating human isogenic diseases models. The three-year technology-access agreement will allow the NCI and NHGRI researchers to generate multiple X-Man™ cell lines annually, for use in both basic research and translational medicine.

Horizon says 18 projects from eight laboratories will initially aim to develop disease models for a range of cancers, chromatin and chromosome function, and cell lines complementary to existing mouse cancer disease models. As part of the deal NCI will in addition act as Center for Excellence in gene editing, and implement a core research group focused on developing disease models for use by its scientists.

In addition, Horizon will work directly with the NHGRI to develop isogenic disease models of melanoma, which it says will extend the range of applications of the Genesis platform within the Genesis Gene Editing Consortium. Last year the firm described its plans to commit resources to making the gene-editing platform available to another 50 academic and not-for-profit research groups over the next five years.

“The establishment of a center of excellence at the NCI/NHGRI is a fundamental pillar in our strategy for this program,” remarks Rob Howes, Ph.D., principal scientist and center of excellence program manager at Horizon. “By bringing together the quality and breadth of research at the NCI/NHGRI with Horizon’s Genesis technology, we will develop disease models that will underpin the research at the NCI and NHGRI.” 

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