NEW YORK – Trace Neuroscience launched on Tuesday, having raised $101 million in a Series A financing round and with a lead therapeutic development program exploring an RNA-based treatment approach for certain patients with amyotrophic lateral sclerosis (ALS).
The Series A funding round was led by Third Rock Ventures, and included investors Atlas Venture, GV, and RA Capital Management.
The South San Francisco, California-based biopharma company is developing genomic medicines for neurodegenerative diseases. In its lead program, Trace is developing an antisense oligonucleotide (ASO) therapy designed to restore production of UNC13A proteins and reestablish healthy communication between nerves and muscle cells. Using this approach, Trace hopes to demonstrate that the ASO can slow disease progression in ALS patients and restore muscle function.
Trace isn't the first drugmaker to explore using ASOs to treat ALS. Biogen in 2023 received approval from the US Food and Drug Administration to market an ASO, Qalsody (tofersen), for a genetic form of ALS specifically caused by mutations in the SOD1 gene. Only about 2 percent of ALS cases are related to SOD1 mutations. According to Trace, its approach has the potential to treat significantly more patients, since roughly 97 percent of people with ALS, including those with sporadic ALS, produce insufficient UNC13A.
The approach is based on discoveries linking abnormal function of the TDP-43 protein with the loss of the UNC13A protein. Abnormal TDP-43, which controls RNA splicing, results in improperly spliced UNC13A mRNA and hinders production of the UNC13A protein.
Trace is betting that its ASO will be able to bind to UNC13A mRNA to regulate its processing and ensure proper splicing. The company is currently investigating potential biomarkers to identify those most likely to benefit from this approach to restoring UNC13A protein expression.
"Our focus is now on rapidly translating this science into a life-changing medicine by advancing our lead program toward the clinic," Trace Cofounder Pietro Fratta, a professor of cellular and molecular neuroscience at the University College London's Francis Crick Institute, said in a statement.
Fratta added that Trace also sees potential in using this approach to treat frontotemporal dementia and some cases of Alzheimer's disease with TDP-43 pathology.