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GEN News Highlights : Jan 6, 2011

Horizon and University of Washington Partner to Apply Gene Editing to IPSCs

$400,000 project aims to generate new X-Man cell lines and bioproduction reagents.

Horizon Discovery has pledged $400,000 in funding for a two-year research collaboration with the University of Washington (UW) that aims to exploit Horizon’s rAAV-based gene-editing technology for engineering human induced pluripotent stem cells (IPSCs) and generating new X-Man™ cell models of genetically defined human cell lines.

The collaboration builds on partnerships established between the UW and Horizon in 2008 and 2009 centered on the gene-editing platform. These deals granted Horizon an exclusive worldwide license to IP relating to the use of parvoviral vectors in human gene editing for cell lines and stem cell applications, and exclusive licenses worldwide for all pharmaceutical, diagnostic, and bioproduction applications across all therapeutic fields.

The latest partnership could enable the generation of thousands of new X-Man cell lines for personalized medicine R&D, together with the development of optimized reagents for producing biologics and ex vivo-derived tissue therapies, Horizon claims. The firm will retain an exclusive right to any resulting new IP.

“The ability to precisely and stably alter the genome of mammalian and especially human cell lines, without introducing errors or exogenous vector sequences, will open up multiple new opportunities for our products and services in pharmaceutical and diagnostic development, as well as bioproduction markets,” comments Darrin M. Disley, Ph.D., Horizon executive chairman. “This latter field has long been subject to compromises in the long-term stability and yield of bioexpressed products, and the genetic stability inherent in stem cells promises big advances in this field.”

Horizon has already used its virally mediated gene-engineering technology, Genesis™, to develop a panel of over 250 genetically defined and patient-relevant X-Man human cell lines, which it claims accurately recapitulate the main genetic mutations underpinning cancers including lung, breast, colorectal, and prostate.These X-Man models are being used by the industry and academic groups worldwide for applications ranging from the identification of new drug targets and drug candidate optimization, to defining patient response genotypes, identifying combinations of drugs to address resistance, and finding new oncology applications for existing drugs.

In December 2010 Horizon confirmed plans to plow new resources into the provision of training and open access to its rAAV Genesis platform for an additional 50 academic and not-for-profit research groups. The move to expand its Genesis Gene Editing Consortium over the next five years forms part of Horizon’s strategy to generate at least 2,500 new X-Man models of cancer, neurodegenerative, and cardiovascular disease.

Existing consortium members include the University of Washington, the University of Torino, Johns Hopkins University, the Translational Genomics Institute, the University of Minnesota, the University of Maryland, Case Western Reserve University and the University of Pittsburgh.