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Nov 8, 2012

Horizon Applies Gene Editing to Huntington’s Disease

  • Horizon Discovery inked a gene-editing research collaboration with the Institute of Neurology, University College London. The partnership will focus on the creation of human isogenic disease model cell lines with insertion of Huntington’s disease-causing triplet repeats. The company will provide UCL with access to GENESIS, its rAAV-mediated human gene-editing platform.

    This collaboration is a novel one for Horizon: It represents its first foray into Huntington’s disease and also the first time that the company has applied its GENESIS technology to insertion of triplet repeats into a wild type genome.

    “Although mouse models have given important insights, it is important when studying human disease to use a broad approach, including cultured cells,” explained Sarah Tabrizi, professor of clinical neurology at the UCL Institute of Neurology, global principal investigator of the TRACK-HD study and leader of all UCL Huntington’s disease projects. “The ability to accurately introduce the triplet repeat mutation into human somatic cell lines offers exciting possibilities in the study of Huntington’s disease and potential therapies.”

    The use of patient-relevant disease models created by Horizon’s rAAV-mediated genome editing technology is well established in oncology, explained Rob Howes, principal scientist, Horizon Discovery. In fact just last month the company established a Center of Excellence for gene editing with Washington University in St Louis and the BRIGHT Institut to translate the genomic data the organizations have generated into disease model cell lines, to advance understanding of cancer.

    The new human isogenic cell lines generated by UCL will be exclusively licensed to Horizon. Horizon will also have an exclusive option to license new intellectual property developed.

    According to Horizon, GENESIS is a highly efficient gene-engineering platform technology. It is a virally mediated homologous recombination technology that is reportedly orders of magnitude more efficient in performing gene-targeting in somatic human cell-types over previous plasmid-based technologies. Horizon is now using this gene-engineering platform to routinely, stably, and precisely engineer a wide-panel of cellular models of genetically defined human disease states, as well as provide their perfectly matched normal genetic backgrounds as a reference.


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