Trace Neuroscience has emerged with $101 million in Series A funding to advance its genetic medicine approach for treating neurodegenerative diseases, with an initial focus on amyotrophic lateral sclerosis (ALS). The financing was led by Third Rock Ventures, with participation from Atlas Venture, RA Capital Management, and GV.
Targeting UNC13A in ALS
Trace Neuroscience is focusing on restoring the function of UNC13A, a protein critical for nerve cell communication. Research indicates that in ALS patients with TDP-43 mutations, UNC13A processing is impaired, leading to nerve cell damage. The company's therapeutic strategy involves an antisense therapy designed to bind to the instructions for UNC13A, ensuring they are properly processed.
"That was the 'aha' moment for us," said Tong, of Third Rock, referring to concurrent discoveries about the ties between UNC13A and TDP-43 made by Gitler’s lab and two others.
Scientific Rationale
TDP-43 is a molecule essential for processing RNA. When TDP-43 mutates, it impairs the production of key proteins, including UNC13A. Studies have linked this dysfunction to nerve-destroying disorders like ALS. Specifically, a deficiency of TDP-43 in nerve cell cores leads to incorrect splicing of genetic instructions for UNC13A, a protein vital for nerve cell communication. UNC13A mutations have been identified as a risk factor for ALS and certain types of dementia.
Clinical Development and Future Plans
Trace Neuroscience anticipates entering human clinical trials with its lead program in early 2026. The initial target indication will be ALS, but the company believes UNC13A restoration could potentially benefit other neurodegenerative conditions as well. Eric Green, CEO of Trace, noted that the company aims to raise enough capital to reach "key clinical data points."
Leveraging Recent Advances in ALS Drug Development
Trace Neuroscience aims to capitalize on the progress made with Biogen's Qalsody, the first antisense therapy approved for ALS patients with specific genetic mutations. The approval of Qalsody has led to the adoption of neurofilament light chain as a key biomarker in ALS drug development, providing Trace with a more robust toolkit for evaluating its molecules.
"That gives us a much stronger toolkit for evaluating our molecules than we had before," Green said.
Leadership and Scientific Team
Trace Neuroscience is led by Eric Green, a former executive at Maze Therapeutics and MyoKardia. The company's co-founders include Aaron Gitler from Stanford University, Pietro Fratta from University College London, and Michael Ward from the National Institutes of Health.
