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Francis Crick Institute and Broken String Biosciences partner for ALS research

The rare and fatal progressive neurodegenerative disease is estimated to affect 5,000 people in the UK.

The Francis Crick Institute and Broken String Biosciences have announced a new collaboration to advance research in amyotrophic lateral sclerosis (ALS).

The partnership aims to advance the understanding of genomic instability in the development of the neurodegenerative disease.

Affecting an estimated 5,000 people in the UK, ALS is a rare and fatal neurological disorder that causes the brain and spinal cord nerve cells to progressively degenerate.

The disorder gradually causes a loss of the ability to control voluntary movements and basic bodily functions.

As part of the collaboration, the research project aims to develop novel applications for Broken String’s proprietary DNA break-mapping platform, INDUCE-seq, to leverage the technology to investigate the impact of genomic instability in the development of ALS.

INDUCE-seq is a scalable platform technology that reveals the breaks induced by any nuclease-based genome editing system with high precision. It was created to address an unmet need for accurate and rapid measurement of off-targets induced by gene editing.

The platform provides data-driven and actionable insights to advance research and development, as well as pre-clinical and clinical gene editing programmes.

Supported by the Crick’s Business Engagement Fund, an initiative supported by the Medical Research Council, Crick researchers will work with the genomics company to demonstrate and further validate the platform in ALS.

Dr Simon Boulton, principal group leader of the Boulton Lab at the Crick, commented: “Our research is focused on exploring how cells repair damage to their DNA and how failures in this process lead to disease.

“We are excited to leverage the INDUCE-seq platform’s unique capabilities… and… deepen our understanding of diseases that have genomic instability as a contributing factor, such as ALS.”

Felix Dobbs, chief executive officer, Broken String, said: “There is an unmet clinical need for effective ALS treatments, as well as strategies for earlier diagnosis that can significantly improve patient outcomes.

“This collaboration… demonstrates a fantastic opportunity to apply our expertise across other key research areas to support the advancement of human health.”