Horizon's Center of Excellence program spans a variety of organizations, consisting of academic and not-for-profit research groups or laboratories to which Horizon makes available resources for training and open access to GENESIS, its proprietary rAAV-mediated human gene- editing platform. The Center of Excellence at Washington University and the BRIGHT Institute will make use of the GENESIS platform to develop isogenic pairs (mutant and wild type) of human cell lines that incorporate genes associated with the development of certain diseases. These cell lines will then be available as accurate disease models to enable additional research. David Piwnica-Worms M.D., Ph.D., Helen Piwnica-Worms, Ph.D., Greg Longmore, M.D., Vijay Sharma, Ph.D., Sheila Stewart, Ph.D. and Jason D. Weber, Ph.D. will serve as the principal investigators for the project.  

 

"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," Dr. Rob Howes, principal scientist at Horizon Discovery, said in a statement. "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 agreement is in keeping with Horizon's goal of generating at least 2,500 new X-MAN (gene X-Mutant And Normal) models within a variety of diseases, including cancer, cardiovascular, neurological and autoimmune diseases. Horizon's X-MAN cell lines "model many of the common genotypes observed in human cancer, plus provide their perfectly matched normal genetic backgrounds as a reference. X-MAN cell lines separate the cancer-gene spectrum to aid the discovery of specifically targeted drugs that can be used singularly or in combination."  

 

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

 

 

SOURCE: Horizon press release