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Horizon Discovery and Washington University in St Louis with the BRIGHT Institute Form Core Facility for rAAV-mediated Genome Editing

17 Oct 2012

World leading genome sequencing institute joins Horizon’s Centers of Excellence program

Cambridge and London, UK, 17 October 2012: Horizon Discovery (Horizon), a leading provider of research tools to support the development of personalized medicines, today announced it has established a Center of Excellence (CoE) for gene editing with Washington University in St Louis and the BRIGHT Institute. One of the leading genome sequencing facilities in the world, Washington University and the BRIGHT Institute will work with Horizon to translate the genomic data they have generated into disease model cell lines, to advance understanding of cancer.

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 new CoE at Washington University and the BRIGHT Institute will use GENESIS to generateisogenic pairs (mutant and wild type) of human cell lines incorporating genes involved in the development of specific diseases, which can then be used as accurate disease models for further research. Principal Investigators for the project are David Piwnica-Worms MD/PhD, Helen Piwnica-Worms PhD, Greg Longmore MD, Vijay Sharma PhD, Sheila Stewart PhD, and Jason D. Weber PhD.

"We are pleased to license to Horizon the human cell lines we have developed at Washington University," says Jason Weber, PhD, associate professor of medicine in the Division of Oncology and researcher at the BRIGHT Institute at Washington University School of Medicine in St. Louis. "With these cell lines, we will use Horizon's GENESIS technology to alter specific genes involved in the development and progression of cancer. We can also test whether existing or investigational drugs are effective against these models of human cancer, an important early step in the development of personalized medicine."

“We are delighted that a genetic research organization of the caliber of Washington University and the BRIGHT Institute has recognized the potential of the GENESIS technology,” commented Dr Rob Howes, Principal Scientist, Horizon Discovery. “We are working with groups around the world to develop an increasing number of cell lines accurately modeling human disease, providing vital tools for understanding, preventing and treating those diseases, and towards more personalized therapies.”

The new human isogenic cell lines generated by Washington University and the BRIGHT Institute will be exclusively licensed to Horizon in return for future product royalties. This forms part of Horizon’s strategy to generate at least 2500 new X-MAN (gene-X, Mutant And Normal) models across a range of disease types including cancer, cardiovascular, neurological and auto-immune diseases. These models support drug discovery researchers in their efforts to understand how complex genetic diseases manifest themselves in real patients, and help rationalize many aspects of drug development, reducing the cost of bringing to market new personalized therapies.

The Centers of Excellence are part of the GENESIS Gene Editing Consortium, which includes rAAV GENESIS pioneers the National Cancer Institute, Cambridge University, Yale University, and Dana-Farber Cancer Institute.