Horizon Discovery (Horizon) today 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. This new cell-model will help researchers understand the effect of miRNA21 in patients and help explore this gene-regulator as a potential new target for therapeutic intervention.
The in-licensed lines will beadded 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.
Dr.Chris Torrance, CSO and co-founder of Horizon said:“Increasingly, non-coding regions of the genome, far from being junk, are being found to have important regulatory roles in gene-expression and cell function. They potentially represent a new class of target for therapeutic intervention and Horizon is delighted to see that rAAV-mediated gene editing has now been applied by Drs. Yu and Zhang at Pittto generate the first clean and stable knock-out model of miR-21. It is our hope that providing such models to the wide research community will speed up the discovery of new drugs to novel cancer targets such as this.”
Dr. Marc S. Malandro, Pitt’s Associate Vice Chancellor forTechnology Management and Commercialization stated: “Using gene-editing technology, Drs. Yu and Zhang have made a valuable contribution to the understanding of miRNA function and the roles miRNAs play in tumorigenesis. These key insights may lead to the development of novel cancer therapies targeting miRNAs. Horizon’s license of the Pitt cell models will help to enable broad availability and use of these important tools for basic research and drug development.”
The license with Pitt is initially for five years and includes up-front fees and an ongoing royalty fees on product sales.