Horizon successfully completes pilot human gene-editing programme with leading pharma

Cambridge Enterprise portfolio company Horizon Discovery (Horizon), has successfully completed an eighteen month pilot programme with leading pharmaceutical company Novartis.

The programme was designed to test the ability of Horizon’s GENESIS rAAV gene-editing platform to generate human isogenic cell lines for use by Novartis in pre-clinical research programmes.

The collaboration between the companies, which began in November 2009, will now be progressed to generate between 30 and 50 new X-MAN (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 modelling 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 programme yielded 12 knock-in and knock-out cell lines across seven 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 UK is the UK affiliate of Swiss based Novartis AG – one of the largest and most widely respected pharmaceutical companies in the world.

Novartis pays Horizon undisclosed fees under this agreement.