Horizon Discovery Secures Exclusive License from University of Pittsburgh to Panel of New Human Isogenic Cell Lines
Panel includes the first human isogenic cell-model for non-codling miRNA gene-regulators
Horizon Discovery announced that it has secured worldwide exclusive rights to a panel of new human isogenic cell models developed by Dr. Jian Yu and Dr. Lin Zhang of the University of Pittsburgh (Pitt) using techniques that are the basis of the Company’s proprietary rAAV GENESIS gene-editing platform. Included in the panel is a novel isogenic cell-model for a key human non-coding RNA gene-expression regulator called miRNA21, which is implicated in a wide range of cancers.
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 X-MAN™ cell lines, which have been referred to as “patients-in-a-test-tube”, accurately model disease-causing mutations. This enables drug 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.
The license with Pitt is initially for five years and includes up-front fees and an ongoing royalty fees on product sales.
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