Horizon Discovery Ltd. and University of Washington Announce Collaboration to Apply rAAV Gene Editing in iPS cells

January 06, 2011 -- Horizon Discovery (Horizon) a leading provider of research tools to support the development of personalized medicines, and the University of Washington (UW) announced today that they have entered into a two-year research collaboration aimed at generating a process for gene editing in inducible human pluripotent stem cells.

The research will exploit the high efficiency and accuracy of rAAV (recombinant Adeno-Associated Virus) gene editing vectors to enable the future creation of thousands of new X-MAN™ (Gene-X; Mutant And Normal) cell models, the world’s first source of genetically-defined and patient-relevant human cell lines. As part of the collaboration, Horizon will fund a two-year $400,000 research program at the University and will have an exclusive right to any new intellectual property created.

The collaboration builds on the agreements signed in April 2009, where the UW granted Horizon an exclusive worldwide license to its patent suite and know-how relating to the use of parvoviral vectors in human gene-editing for in-vitro human cell-lines and stem cell applications. This followed on from a previous agreement, signed in 2008, whereby the UW granted Horizon exclusive worldwide licenses for all pharmaceutical, diagnostic and bio-production applications across all therapeutics fields of use.

The research program, which will be led by Professor David Russell, the inventor of rAAV gene-editing, and who has already demonstrated the concept of targeting human pluripotent stem cells1, will enable the generation of diverse arrays of tissue derived disease models for personalized medicine R&D applications, as well as optimized reagents for the production of biological drugs/materials and ex-vivo-derived tissue therapies.

Commenting on the collaboration, Dr Darrin M Disley, Executive Chairman of Horizon, said: “Our relationship with the University of Washington is a central force in the development of our company. The new agreements will further align the technical and commercial interests of Horizon and UW. 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. This latter field has long been subject to compromises in the long-term stability and yield of bio-expressed products and the genetic stability inherent in stem cells promises big advances in this field.”

Commenting on the announced collaboration with Horizon, Linden Rhoads, Vice Provost of UW Center for Commercialization said, “Horizon has been a partner and research sponsor since 2007. This new joint project furthers our strong work on in vivo gene-therapy applications using AAV by leveraging Horizon’s expertise in cancer genetics and drug discovery with UW AAV-research. We expect to accelerate progress in developing novel in vitro disease models which can capitalize on genome sequencing efforts to foster new drug discovery.”

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