Horizon Discovery licenses two gene-editing technologies

Company boosts rAAV gene-editing platforms with technologies from Sigma Life Science and Harvard

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ST. LOUIS—Sigma-Aldrich Corp.'s Sigma Life Science unit, its biological products and services business, has entered into a non-exclusive license agreement with Horizon Discovery for use of Sigma's CompoZr Zinc Finger Nuclease (ZFN) technology. The agreement gives Horizon the commercial freedom to apply the technology alongside its proprietary rAAV technology in its gene-editing service and cell line product businesses. Under the terms of the deal, Horizon will obtain CompoZr ZFNs manufactured by Sigma Life Science for use in commercial development of cell-based products and to provide commercial gene editing services. Financial terms were not disclosed.  
Horizon will immediately begin applying the CompoZr (pronounced "composer") ZFN technology as part of its custom cell-line generation service along with its own rAAV technology, to efficiently and cost-effectively generate a number of novel gene-edited cell lines. The offering will be marketed under Horizon's precision gene-editing brand, GENESIS.
"Licensing CompoZr ZFN technology to Horizon is another step in making the most robust and flexible gene-editing technology widely available to the entire research community," says Dr. Paul Brooks, global market segment manager at Sigma-Aldrich. "We are pleased that Horizon will be using its expertise to apply both rAAV and CompoZr ZFN gene-editing technologies side by side for the benefit of the research community."  
Brooks notes that ZFN provides the big benefit of targeting all the copies of a single gene—all four alleles, for example, will be mutated, while with rAAV the process produces one allele at a time. To sum up, Brooks states that Horizon will use ZFN "to do things they can't do with rAAV."  
"Nuclease-based approaches are particularly adept at achieving rapid and efficient knock-out of two or more alleles, making them a perfect complement to Horizon's proprietary rAAV-based precision gene-editing platform," agrees Eric Rhodes, chief technology officer at Horizon, who has 15 years of experience in ZFN and rAAV gene-editing technology. "Our scientists have the expertise and now an unrivalled toolset to help guide customers toward the approach that best answers their biological question and/or suits their timescale and budget."  
In a second move aimed at expanding its gene-editing base, Horizon announced it has entered into a non-exclusive license agreement with Harvard University to access intellectual property related to the commercialization of CRISPR gene-editing technology for research use. By adding CRISPR and ZFN to its GENESIS precision gene-editing platform, Horizon claims it can now offer researchers an unrivalled toolbox capable of performing rapid functional genomics experiments, as well as creation of high-precision human disease models for deployment at all stages of drug discovery and diagnostic development. Horizon will employ all three genome-editing technologies for custom client-led projects, as well as to expand on its own menu of more than 500 genetically defined off-the-shelf cell lines and related products. In addition, the company will soon launch a range of rAAV, CRISPR and hybrid rAAV/CRISPR gene-editing kits and associated reagents, supported by Horizon's technical team with expertise in all gene-editing platforms and their application in translational research.  
CRISPR is an RNA-guided gene editing system that can introduce either a targeted double strand break or single strand nick in the genome of mammalian cells.  
"The introduction of a nick rather than a full double strand break offers advantages over other nuclease technologies when the goal of the project is to introduce a specific mutation rather than simply disrupting the gene," Rhodes says. "By combining both CRISPR and ZFNs with our proprietary rAAV technology, Horizon is working to develop novel approaches that achieve levels of gene-editing efficiency not previously seen when using a single approach alone."  

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