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Horizon, UCL Collaborate on Huntington's Disease Research

NEW YORK (GenomeWeb News) – Horizon Discovery said today it has entered into a research collaboration covering gene editing with the Institute of Neurology, University College London.

The partnership will focus on creating human isogenic disease model cell lines with the insertion of Huntington disease-causing triplet repeats. The program at UCL is a new disease area for Horizon, and inserting triplet repeats into a wild type genome is a new alteration application for the company's rAAV-mediated human gene-editing platform called Genesis, it said.

Horizon will provide training and access to Genesis.

The new cell lines generated by UCL will be licensed exclusively to Horizon, which will also have an exclusive option to license new IP developed from the collaboration. The deal also furthers the Cambridge, UK-based firm's goal to generate at least 2,500 new X-MAN (gene x-Mutant And Normal) models of cancer, neurodegenerative, and cardiovascular disease.

Financial and other terms of the deal were not disclosed.

"The use of patient-relevant disease models created by Horizon’s rAAV-mediated genome editing technology is well established in oncology,” Rob Howes, principal scientist at Horizon, said in a statement. "We believe that by developing the application of this technology to other disease areas such as Huntington’s disease, we can provide a vital tool for understanding, preventing, and treating those diseases."