Cell line technology from Cambridge is at the heart of a new UK alliance designed to develop personalised therapies for Huntington’s disease.

Horizon Discovery has joined forces with the Institute of Neurology, University College London (UCL) for the initiative, which leverages its cancer gene-editing prowess for research into other disease areas.

Huntington’s disease (HD) is a neurodegenerative genetic disorder that affects muscle coordination and leads to cognitive decline and psychiatric problems.

Horizon, which provides research tools to support the development of personalised medicines, says the UCL programme will focus on the creation of human isogenic disease model cell lines with insertion of Huntington’s disease-causing triplet repeats.

The collaboration contributes to an academic not-for-profit research group to which Horizon commits resources to provide training and access to its proprietary rAAV-mediated human gene-editing platform, GENESIS™.

The programme at UCL represents a new disease area for Horizon and the insertion of triplet repeats into a wild type genome is a novel genetic alteration application for the GENESIS technology.

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, has no doubts about the significance of the new programme.

Professor Tabrizi said: “Although mouse models have given important insights, it is important when studying human disease to use a broad approach, including cultured cells.

“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.”

And Dr Rob Howes, principal scientist at Horizon Discovery added: “The use of patient-relevant disease models created by Horizon’s rAAV-mediated genome editing technology is well established in oncology.

“We believe that by developing the application of this technology to other disease areas such as Huntington’s, we can provide a vital tool for understanding, preventing and treating those diseases.”

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.

This forms part of Horizon’s strategy to generate at least 2500 new X-MAN™ (gene X- Mutant And Normal) models of cancer, neurodegenerative, and cardiovascular disease.

These models support drug discovery researchers in their efforts to understand how complex genetic diseases manifest themselves in real patients, and help rationalise many aspects of drug development, reducing the cost of bringing to market new personalised therapies.


Source: Business Weekly

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