Trace Medicine has launched with $101 million in series A funds and a mission to treat amyotrophic lateral sclerosis (ALS) by targeting the same protein that recently caught the attention of Eli Lilly.
Almost all cases of ALS and half of frontotemporal dementia (FTD) cases are linked to dysfunction in a protein TDP-43. Academic research, including at Stanford University, in the past couple of years has also appeared to confirm that the lack of TDP-43 could sabotage the proper expression of an important neuronal protein called UNC13A.
South San Francisco-based Trace has been formed to take forward these discoveries, with Aaron Gitler, Ph.D., professor of genetics at Stanford, among the academic heavyweights who have co-founded the biotech.
“Genomic-based therapies have begun to transform the lives of people living with ALS,” Gitler explained in a Nov. 12 release. “But so far, they have only been effective for those rare forms of the disease caused by SOD1 or FUS mutations, which account for only 3% of all ALS cases. The remaining people, including those with sporadic disease, the most common form that occurs randomly without a clear cause, need new treatments grounded in human genetics with defined mechanisms of action.”
Trace is working on genomic therapies that restore UNC13A to reestablish connections between nerves and muscle cells that have been affected by ALS. The biotech’s lead program is an antisense oligonucleotide (ASO) that aims to “preserve and potentially improve muscle function” in ALS patients, including the sporadic form of the disease.
The company has secured $101 million to take forward these ambitions via a series A fundraise led by Third Rock Ventures, with Atlas Venture, GV and RA Capital Management all getting involved.
“UNC13A is a highly compelling genetic target directly linked to ALS disease progression and survival,” Trace’s CEO Eric Green, M.D., Ph.D., said in the release. “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.”
While the initial focus is on ALS, the company also sees potential to move into other brain diseases that involve the loss of UNC13A such as FTD and Alzheimer’s.
Trace’s team members aren’t the only ones who have spied potential in restoring UNC13A. In June, Lilly paid QurAlis $45 million for a preclinical splice-switching ASO designed to use the same method to treat FTD and ALS, while AcuraStem has an ASO against UNC13A in development for ALS and FTD.