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. "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.
Horizon recently launched an online support site, www.rAAVers.org, for scientists working with rAAV-mediated genome editing.
About Horizon Discovery http://www.horizondiscovery.com/
Horizon Discovery Limited (Horizon) is a leading provider of research tools to support translational genomics research and the development of personalized medicines. The Company’s proprietary rAAV gene-editing technology, GENESIS™, is industry leading. Using GENESIS, Horizon is able to alter any endogenous gene sequence of a human or mammalian cell-line quickly, reliably and without introducing unwanted and confounding genotypes and/or phenotypes.
Horizon has applied GENESIS to create over 400 X-MAN™ cell lines, the world’s first source of genetically-defined and patient-relevant human cell lines, accurately modeling the disease-causing mutations found in cancer patients. These ‘patients-in-a-test-tube’ are being used by academic and industry leaders to identify the effect of individual or compound genetic mutations on drug activity, patient responsiveness, and resistance, leading to the successful prediction of which patient sub-groups will respond to currently-available and future drug treatments. This enables the design of shorter, more focused, and less expensive clinical trials, ultimately providing the tools to identify the ‘right drugs’ for the ‘right patients’ based upon the unique genetic mutations that define their disease.
In addition to the X-MAN cell lines, Horizon provides GENESIS and X-MAN™ derived products and services, with industrial application in: bio-pharmaceutical process optimization; clinical diagnostic development; drug discovery & development; and the provision of reference standards for genomic-based clinical research platforms.
GENESIS and X-MAN are registered trademarks of Horizon Discovery Limited.
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Contacts:
Media enquiries:
Katie Odgaard
Zyme Communications
Tel: +44 (0)7787 502 947
Email katie.odgaard@zymecommunications.com
At Horizon:
Dr Rob Howes
Principal Scientist
Tel: +44 (0) 1223 655 584
Email: r.howes@horizondiscovery.com
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