Horizon successfully completes pilot human gene-editing program

Horizon Discovery (Horizon), a leading provider of research tools to support the development of personalized medicines,has announced that it has successfully completed an eighteen month pilot program with Novartis, designed to test the ability of Horizon’s GENESISTM rAAV gene-editing platform to generate human isogenic cell lines for use by Novartis pre-clinical research programs. The collaboration between the companies, which began in November 2009, will now be progressed to generate 30-50new X-MANTM (gene X- Mutant And Normal) cell lines annually.

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 switching on a natural and 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 correction of genetic defects in gene therapy applications, or the accurate modeling of genetic diseases in human cells in vitro.

Dr Chris Torrance, Chief Scientific Officer of Horizon, said: “This is a significant achievement as Novartis challenged the length and breadth of performance of our rAAV gene-editing technology. The program yielded 12 knock-in and knock-out cell lines across 7 parental cell-lines, including three that had not been targeted before. A number of the completed projects demonstrated new application of our technology in developing functional genomics tools that could be deployed for target rescue/validation and early drug discovery research”.

Novartis pays Horizon undisclosed fees under this agreement.