Horizon, WUSTL establish Center of Excellence

CAMBRIDGE, United Kingdom—Horizon Discovery has announcedthe establishment of a Center of Excellence for gene editing together withWashington University in St. Louis and the BRIGHT Institute (Bridging Researchwith Imaging, Genomics and High-Throughput). The BRIGHT Institute, part of theuniversity's cancer efforts, is "dedicated to mechanism- and discovery-basedscience and structured to enable rapid translation of breakthroughs intopatient care through molecular imaging, function genomics and high-throughputtechnologies." Representing one of the premier genome sequencing facilitiesworldwide, Washington University and the BRIGHT Institute will work alongsideHorizon to translate their genomic data into disease model cell lines for theadvancement of cancer knowledge. The new cell lines will be exclusivelylicensed to Horizon in return for future product royalties.

"We are pleased to license to Horizon the human celllines we have developed at Washington University," Jason Weber, Ph.D.,associate professor of medicine in the Division of Oncology and researcher atthe BRIGHT Institute at Washington University School of Medicine in St. Louis,said in a press release. "With these cell lines, we will use Horizon'sGENESIS technology to alter specific genes involved in the development andprogression of cancer. We can also test whether existing or investigationaldrugs are effective against these models of human cancer, an important earlystep in the development of personalized medicine."

Horizon's Center of Excellence program spans a variety oforganizations, consisting of academic and not-for-profit research groups orlaboratories to which Horizon makes available resources for training and openaccess to GENESIS, its proprietary rAAV-mediated human gene-editing platform.The Center of Excellence at Washington University and the BRIGHT Institute willmake use of the GENESIS platform to develop isogenic pairs (mutant and wildtype) of human cell lines that incorporate genes associated with thedevelopment of certain diseases. These cell lines will then be available asaccurate disease models to enable additional research. David Piwnica-WormsM.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 theprincipal investigators for the project.

"We are delighted that a genetic research organization ofthe caliber of Washington University and the BRIGHT Institute has recognizedthe potential of the GENESIS technology," Dr. Rob Howes, principal scientist atHorizon Discovery, said in a statement. "We are working with groups around theworld to develop an increasing number of cell lines accurately modeling humandisease, providing vital tools for understanding, preventing and treating thosediseases, and towards more personalized therapies."

The agreement is in keeping with Horizon's goal ofgenerating at least 2,500 new X-MAN (gene X-Mutant And Normal) models within avariety of diseases, including cancer, cardiovascular, neurological andautoimmune diseases. Horizon's X-MAN cell lines "model many of the commongenotypes observed in human cancer, plus provide their perfectly matched normalgenetic backgrounds as a reference. X-MAN cell lines separate the cancer-genespectrum to aid the discovery of specifically targeted drugs that can be usedsingularly or in combination."

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

SOURCE: Horizon press release