Broken String Biosciences, a gene therapy company based in Cambridge, U.K., has announced a new research collaboration with the Francis Crick Institute in London. The Crick Institute’s researchers plan to leverage Broken String’s DNA break-mapping platform, called INDUCE-seq™, to develop novel applications for amyotrophic lateral sclerosis (ALS) therapies.

ALS, also known as Lou Gehrig’s disease, is a progressive, incurable neurodegenerative disease affecting voluntary control of the body and basic bodily functions. Over 90% of ALS cases are sporadic and little is known about the underlying causes. Most treatment options focus on symptom management and stalling disease progression, however, deeper understanding of the genetics causing ALS would open the door to more personalized therapies.

Researchers at the Francis Crick Institute, a biomedical discovery institute dedicated to understanding the fundamental biology underlying health and disease, have been working on understanding the underlying mechanism of ALS disease mechanism. According to Simon Boulton, PhD, his lab at the Crick Institute has been “exploring how cells repair damage to their DNA, and how failures in this process lead to disease.” Another Crick team, led by Rickie Patani, PhD, has been working to illuminate the underlying mechanisms of ALS disease progression. Together, they are focused on determining the role of genome stability in ALS development.

“We are excited to leverage the INDUCE-seq platform’s unique capabilities in directly measuring and quantifying DNA double-strand breaks, and applying this to deepen our understanding of diseases that have genomic instability as a contributing factor, such as ALS,” Boulton said.

Broken String is a genomics company focused on advancing the accessibility of more precise, safe, and effective cell and gene therapies through its technology platforms. The company’s primary platform, INDUCE-seq, is driven by next-generation sequencing methods to measure and quantify off-target gene edits. It provides data for a variety of applications from discovery, to preclinical and clinical therapy development stages.

“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,” said Felix Dobbs, PhD, CEO, Broken String Biosciences.

Ultimately, they hope their collaboration will translate into new diagnostic and treatment options for ALS patients. Dobbs added, “There is an unmet clinical need for effective ALS treatments, as well as strategies for earlier diagnosis that can significantly improve patient outcomes. We look forward to working closely with Dr. Boulton and Professor Patani’s groups to support this critical research area and continue building out our application focuses.”

Broken String will be sharing more information about their technology at their booth at ASGCT (#435) and they will be presenting a poster (1682) on Friday, May 10 at 12pm entitled, “INDUCE-seq: Ensuring the Safe Development of Cell and Gene Therapies by Gene Editing.”

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