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New Biopharmaceutical Company to Work on UNC13A Protein Cure to ALS
The startup has secured $101 million in funding to pursue a treatment that could impact 97% of ALS patients.

November 15, 2024 by Laurie Watanabe

Trace Neuroscience, a biopharmaceutical launch based in South San Francisco, is targeting the UNC13A protein as a possible treatment path for amyotrophic lateral sclerosis (ALS) and other neurogenerative diseases.

In a Nov. 12 press release, the company said UNC13A “is a genetically validated target with the potential to treat 97% of people living with ALS.”

Trace Neuroscience said it’s working with $101 million in start-up funding from Atlas Venture, GV and RA Capital Management.

“The company is developing novel genomic therapies that restore UNC13A protein to reestablish healthy communication between nerves and muscle cells impacted by neurodegenerative disease,” the press release said.

“UNC13A is a highly compelling genetic target directly linked to ALS disease progression and
survival,” said Eric Green, M.D., Ph.D., co-founder/CEO of Trace Neuroscience. “Insights from the human genome have led to transformative medicines for many diseases, and with what we now know about the role of UNC13A, we believe the time is right to apply this approach to ALS. We envision a world where UNC13A restoration improves outcomes across a range of neurodegenerative diseases, including for the approximately 30,000 people in the U.S. living with ALS.”

“UNC13A is critical for neurons to communicate amongst each other and with muscles through
synaptic function, which is lost in ALS,” said Trace Neuroscience co-founder Pietro Fratta, M.D., Ph.D., professor of cellular and molecular neuroscience at University College London, Francis Crick Institute. “Being able to re-establish this is groundbreaking. This is an exciting time in brain disease innovation, and we also see potential for treating frontotemporal dementia and over half of Alzheimer’s disease patients where TDP-43 pathology occurs and UNC13A is lost.”

In addition to Green and Fratta, Trace Neuroscience co-founders include Aaron Gitler, Ph.D., professor of genetics, Stanford University; and Michael Ward, M.D., Ph.D. senior investigator, National Institute of Neurological Disorders and Stroke, National Institutes of Health.

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