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Horizon Secures License from Case Western to 35 Endogenously Tagged Human Isogenic Cell Lines

09 Dec 2010

Horizon Discovery (Horizon) today announced it has secured worldwide exclusive rights to a panel of new human isogenic cell models developed by Dr. Zhenghe Wang, Assistant Professor of Genetics at Case Western Reserve University School of Medicine, using its proprietary rAAV GENESIS gene-editing platform.

The in-licensed lines will be added to Horizon’s rapidly expanding library (300+) of X-MANTM (gene X- Mutant And Normal) cell models, which are the world’s first source of genetically-defined and patient-relevant human cell lines.These cell lines, which have been referred to as “patients-in-a-test-tube”, accurately model disease-causing mutations. This enablesdrug discovery researchers to understand how cancer manifests itself in real patients and identify the effect of individual mutations on drug activity, patient responsiveness and resistance. X-MAN cell lines are also being used to successfully predict which patient sub-groups will respond to currently-available and future drug treatments, helping to rationalize many aspects of drug development; and therefore the final cost of new personalized cancer therapies.

At the heart of the GENESIS platform is the use of rAAV vectors that have a unique and powerful property in performing accurate and efficient gene-editing functions in human cells by exploiting homologous recombination (HR). When harnessed using rAAV gene-editing vectors, HR allows the precise alteration of any DNA sequence, permitting the correction of genetic defects in gene therapy applications, or the accurate modeling of genetic diseases in human cells in vitro.

Dr. Chris Torrance, CSO and co-founder of Horizon said: “We are constantly looking for new applications for GENESIS; our ubiquitous and accurate human gene editing platform. Precisely tagging specific endogenous proteins at their physiological expression levels, without manifesting unwanted off-target sequence errors, is a new and important application. Using these new cell-lines, researchers will be able to decipher how many disease-associated genes work by causing new networks of protein-protein interactions to occur in a cell; and will open up new routes to develop novel targeted therapies”.

Dr. Stacy Fening of Case Western Reserve University’s Technology Transfer Office stated: “In partnering with Horizon, we gain a tremendous opportunity to bring these custom cancer cell lines to the market. Dr. Wang is an innovative researcher and we are pleased to be able to offer his cells among Horizon’s library. Commercial deployment of these cells allows them to be used to their full potential to advance academic and pharmaceutical research in the cancer field.”

Dr. Darrin M Disley, Executive Chairman of Horizon stated, “I am delighted that we are able to provide a commercial outlet for Dr. Wang’s unique cell-lines. The launch of a panel of 35 X-MANTAG lines will enable our customers to better understand the endogenous proteins that are activated / expressed when a human cell picks-up the mutations implicated in the onset and progression of human cancers”.

The license is initially for ten years and includes up-front fees and an ongoing royalty on product sales.