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Horizon, Washington University and the BRIGHT Institute Form Core Facility for rAAV-Mediated Genome Editing

Published: Thursday, October 18, 2012
Last Updated: Thursday, October 18, 2012
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World leading genome sequencing institute joins Horizon’s CoE program.

Horizon Discovery (Horizon) has announced that 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 generate isogenic 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.

Weber continued, "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.

Dr Howes continued, “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.

Horizon recently launched an online support site,, for scientists working with rAAV-mediated genome editing.

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