Broken String Biosciences has teamed up with the Francis Crick Institute to conduct research into the role of genome stability in amyotrophic lateral sclerosis (ALS). 

The aim of the project is to expand the applications for Broken String’s core technology, a DNA break mapping platform named Induce-seq. The platform is particularly focused on mapping off-target gene edits, allowing manufacturers to measure the unintended impacts of their therapies more accurately. The research will be focused on leveraging the technology to investigate the impact of genomic instability in the development of ALS.  

The partnership has been secured via the Francis Crick Institute’s Business Engagement Fund; a new initiative supported by The Medical Research Council (MRC-UKRI). The initiative is designed to encourage collaborations with small and medium-sized enterprises (SMEs) and strengthen the Crick’s engagement with industry. 

In September 2023, Broken String Biosciences acquired a $15m investment in its Series A funding round, with Illumina Ventures as the key industry investor. Illumina Ventures is the investment wing of biosciences giant Illumina. 

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to muscle weakness, paralysis, and eventually respiratory failure. The ALS research landscape has proven challenging over the last few years. There are only two approved drugs on the market, Sanofi’s riluzole and Mitsubishi’s Radicava (edaravone). Both drugs are designed to manage the disease but are not curative, meaning there is an urgent need to move towards disease-modifying therapies. 

This challenge was highlighted earlier this year, when Amylyx Pharmaceuticals withdrew its ALS drug Relyvrio (AMX0035) from the market following disappointing results from a Phase III confirmatory trial. The Phoenix trial failed to meet any of its endpoints, causing the company’s stock to plummet by more than 85%. Relyvrio, which received US Food and Drug Administration (FDA) approval in September 2022, is now unavailable except to patients who can continue taking it as part of a free drug program.  

In the announcement accompanying the research collaboration, Broken String Bioscience’s CEO Felix Dobbs said: “This collaboration with the Crick Institute is validation of our differentiated approach to DNA break-mapping; enabling our team to support world-leading research with insights provided through our INDUCE-seq platform. It demonstrates a fantastic opportunity to apply our expertise across other key research areas to support the advancement of human health.”