The agreement will enable the NIH CRM to apply Horizon’s GENESIS™ genome editing technology to engineer stem cells in order to create isogenic disease models with induced mutations and/or lineage markers. These disease models will be used to help researchers understand the effects of specific genes and mutations as stem cells differentiate, as well as create lineage reporters for stem cell differentiation.
The NIH CRM was recently established through support from the NIH Common Fund to provide the infrastructure to support and accelerate the clinical translation of stem cell-based technologies, and to develop widely available resources to be used as standards in stem cell research.
The GENESIS technology harnesses rAAV vectors to perform accurate and efficient gene-editing functions in human cells, by switching on a natural high-fidelity DNA-repair mechanism called homologous recombination (HR). When harnessed using rAAV gene-editing vectors, HR allows the precise alteration of any DNA sequence, permitting the accurate modeling of genetic diseases in human cells, including stem cells, in vitro. The use of these models in oncology is well established, and has predicted patient responses to targeted therapies both during drug development and in the clinic. By applying this technology to other scientific areas Horizon hopes to lead to the development of personalized medicines by advancing scientific research.
“Through our CoE program, we aim to work with world leading experts in their fields, so we are delighted to be working with the NIH CRM, a key centre for stem cell research,” said Dr Rob Howes, CoE Program Manager, Horizon Discovery. “This agreement builds on proof of concept work carried out by Horizon Scientific Advisory Board member and rAAV-mediated gene editing inventor, David Russell, demonstrating the ability of rAAV to gene target in ES cells, and extends the scope of the CoE program into stem cell research.”
Horizon’s CoE programme 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™. Horizon also 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:
At Horizon:
Dr Rob Howes
CoE Program Manager
Tel: +44 (0) 1223 655 584
Email: r.howes@horizondiscovery.com
Media enquiries:
Katie Odgaard
Zyme Communications
Tel: +44 (0)7787 502 947
Email katie.odgaard@zymecommunications.com
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