Trace Neuroscience – a biopharmaceutical company expanding the promise of genomic medicine for people living with neurodegenerative diseases – announced its launch with a $101 million Series A funding led by Third Rock Ventures, with participation from Atlas Venture, GV, and RA Capital Management. And the company is developing novel genomic therapies that restore UNC13A protein to re-establish healthy communication between nerves and muscle cells impacted by neurodegenerative disease.
Trace Neuroscience’s lead program is an antisense oligonucleotide (ASO) designed to preserve and potentially improve muscle function in people with amyotrophic lateral sclerosis (ALS), including those with the sporadic form that affects nine out of 10 people with the disease.
The formation of Trace Neuroscience is based on simultaneous convergent discoveries from the company’s co-founders connecting abnormal function of TDP-43 protein with the loss of UNC13A protein, an essential component for neuronal communication in the brain and spinal cord. In people with ALS, this relationship progressively usually breaks down and leads to decreased mobility, paralysis and difficulty breathing.
About 97% of people with ALS produce insufficient amounts of UNC13A, which is directly regulated by the TDP-43 protein that controls RNA splicing. And when TDP-43 stops functioning normally, as occurs in nearly all people with ALS, the UNC13A messenger RNA (mRNA) is improperly spliced, which hinders adequate UNC13A production. Trace Neuroscience’s ASO development candidate was designed to deliver a targeted intervention by binding directly to UNC13A mRNA to regulate its processing and ensure proper splicing, thereby correcting synaptic dysfunction and preserving neuronal signaling.
Trace Neuroscience leadership includes clinicians at the forefront of neurodegenerative research, renowned neuroscience drug developers and proven company builders such as co-founders Pietro Fratta, M.D., Ph.D., Professor of Cellular and Molecular Neuroscience, University College London, Francis Crick Institute; Aaron Gitler, Ph.D., Professor of Genetics, Stanford University; Eric Green, M.D., Ph.D., CEO, Trace Neuroscience; and Michael Ward, M.D., Ph.D., Senior Investigator, National Institute of Neurological Disorders and Stroke, National Institutes of Health. It also includes the management team Irina Antonijevic, M.D., Ph.D., Chief Medical Officer; Megan Baierlein, Chief Operating Officer; Eric Green, M.D., Ph.D., CEO; Marjie Hard, Ph.D., SVP, Development Sciences; and Nick Mordwinkin, Pharm.D., Ph.D., Chief Business Officer and Chief Strategy Officer; Precillia Redmond, Chief People Officer. The board of directors includes Jeffrey Tong, Ph.D., Chairman; Partner, Third Rock Ventures; David Grayzel, M.D., Partner, Atlas Venture; Eric Green, M.D., Ph.D., CEO, Trace Neuroscience; Douglas Kerr, M.D., Ph.D., Chief Medical Officer, Dyne Therapeutics; Anthony Philippakis, M.D., Ph.D., General Partner, GV; Richard Scheller, Ph.D., Chairman of R&D, BridgeBio; and Dodzie Sogah, Ph.D., Venture Partner, Third Rock Ventures
KEY QUOTES:
“UNC13A is a highly compelling genetic target directly linked to ALS disease progression and survival. 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.”
- Eric Green, M.D., Ph.D., co-founder and CEO of Trace Neuroscience
“Genomic-based therapies have begun to transform the lives of people living with ALS. 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. Our insights into UNC13A biology may be the key to slowing disease progression, preserving or restoring muscle function and extending survival for people living with ALS.”
- Co-founder Aaron Gitler, Ph.D., Professor of Genetics at Stanford University
“UNC13A is critical for neurons to communicate amongst each other and with muscles through synaptic function, which is lost in ALS. Being able to re-establish this is groundbreaking. Our focus is now on rapidly translating this science into a life-changing medicine by advancing our lead program toward the clinic. 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.”
- Co-founder Pietro Fratta, M.D., Ph.D., Professor of Cellular and Molecular Neuroscience at University College London, Francis Crick Institute
“The Trace Neuroscience team is strongly positioned with the right leadership, the right scientific approach and the right molecules to disrupt the treatment paradigm in ALS and beyond. People living with neurodegenerative diseases are waiting for more effective new treatments, and UNC13A restoration has the potential to deliver meaningful innovation that will improve their quality of life and overall health outcomes.”
- Chairman Jeffrey Tong, Ph.D., Partner at Third Rock Ventures
