The Cambridge, UK-based research company has its own recombinant adeno-associated virus (rAAV) based genome technology but has entered a license agreement with Sigma-Aldrich (SA) to add the CompoZr ZFN tech as part of its cell line generation service.
“Both rAAV and nuclease-based technologies have their strengths and limitations, and so are best applied in different situations,” Horizon’s CEO, Dr. Darrin Disley told Biopharma-Reporter.com.
ZFNs are particularly adept to making ‘knockouts’ – a technique to make genes inoperative in order to research genes or to understand the mechanism of a specific drug’s action - quickly and inexpensively, he explained, though suffer from several limitations including the fact not all cell lines are transfectable with the tech.
However, Horizon’s rAAV platform, though slower and may be more costly, “is based on homologous recombination (the means by which cells perfectly repair genes that are damaged and so no double-stranded breaks are required,” he added.
“Being in the unique position of having access to both technologies, Horizon is also now working towards merging rAAV with nucleases (deploying them separately and together) to create the next generation of gene editing tools… the combination of rAAV (unique to only Horizon) and ZFN promise a big step forward in gene editing performance.”
The deal with SA, of which financial details were not divulged, is on a non-exclusive basis, though Disley told us he did not expect SA to be making any similar agreements.
Horizon will offer its customers cell line generation services for the routine modification of host mammalian genomes using this joint technology, while – the company says - maintaining cell line stability for improvements in selection, yield and timelines for biomanufacturing.
Genetically-Defined Cell Lines Demand
The ENCODE (Encyclopedia Of DNA Elements) project launched by the National Human Genome Research Institute (NHGRI) in 2003 has suggested up to 80% of genetic variation has biochemical function instead of, Disley said. It was historically thought 2% of genetic variation was functionally relevant and so demand from researchers for genetically-defined cell lines is increasing.
“This demand is going to be driven exponentially in years to come with cost, time and efficacy of gene-editing products/services reduce.”