Maze Therapeutics Announces Publication in Nature Describing a Novel Mechanism Linking Well-Established Genetic Drivers of ALS and FTD

Findings Highlight Applicability of Maze’s Compass Platform to Identify and Provide Insight into Disease-Causing Genetic Variants to Treat Patients in Need

Excerpt from the Press Release:

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Maze Therapeutics, a company translating genetic insights into new precision medicines for patients, today announced the publication in Natureof new findings describing a connection between genetic variation in UNC13A, a gene critical to the function of the nervous system, and the abnormal build-up of TDP-43, a hallmark molecular characteristic of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The research was led by Maze Therapeutics’ co-founder Aaron Gitler, Ph.D., at Stanford University in collaboration with researchers at Maze Therapeutics and other institutions, including Mayo Clinic, UCSF, University of Pennsylvania and Johns Hopkins University. The findings help explain how a genetic variant of UNC13A and dysfunctional TDP-43 can lead to the development of ALS or FTD.

“The findings published today fill a long-standing gap in the understanding of how ALS and FTD develop, two serious neurodegenerative diseases that have no known cures or broadly effective treatments,” said Eric Green, M.D., Ph.D., senior vice president, research & translational sciences. “UNC13A and TDP-43 are well-established contributors to ALS, but how they lead to disease had been a mystery. This study provides the first evidence that these genes act through a common mechanism. Our Compass platform is built to find genetic insights like this, which is a key step in translating them into therapies for patients. This is an exciting step forward for the field as we continue to explore novel approaches targeting TDP-43 pathology.”

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