Trace Neuroscience, a new biotech startup, has emerged with $101 million in funding to develop genetic medicines for amyotrophic lateral sclerosis (ALS). The company aims to address a critical unmet need in ALS treatment by targeting the disease's underlying genetic mechanisms.
Novel Approach to ALS Treatment
Trace Neuroscience's approach is rooted in recent discoveries from the labs of Aaron Gitler (Stanford University), Pietro Fratta (University College London), and Michael Ward (NIH). These labs identified connections between RNA-processing molecules and proteins often impaired in ALS and other neurodegenerative disorders. This foundational science aims to develop treatments that could potentially benefit a broad ALS patient population.
Eric Green, CEO of Trace, emphasized the importance of focusing on robust human genetics to guide their therapeutic development. "Here is this focus on robust human genetics as our guide to give us confidence we're going after a mechanism relevant in people with ALS, rather than in some cell or animal model of the disease," Green stated. The company believes that understanding the mechanism by which the gene is working is critical for therapeutic intervention.
Leveraging Antisense Oligonucleotide Technology
Trace Neuroscience is utilizing antisense oligonucleotides, a technology similar to that used in Biogen's Qalsody (tofersen). Antisense oligonucleotides regulate protein production by binding to RNA. Qalsody received approval based on its effects on neurofilament light chain, a biomarker associated with nerve cell damage, despite not meeting its primary endpoint in clinical trials. Trace plans to incorporate these learnings into its development strategy.
Green noted that the experience with Qalsody has provided valuable insights into biomarker utilization and clinical trial design. "A lot of the things they learned with respect to biomarkers, in particular, are very relevant for how we think about our clinical development," he said. Trace aims to use biomarkers to accelerate proof-of-concept studies in the clinic.
Development Timeline and Investor Confidence
Trace Neuroscience is on track to begin human testing in early 2026. The company's single-asset focus allows it to concentrate resources on achieving meaningful clinical data. Investors, including Third Rock Ventures, Atlas Venture, RA Capital Management, and GV, are confident in Trace's approach due to its strong genetic foundation, well-defined mechanism of action, and the potential for rapid clinical development.
Jeffrey Tong, a partner at Third Rock Ventures and a Trace board member, highlighted the precedent set by Qalsody as a factor in their investment decision. The ability to leverage biomarker data and accelerate clinical trials has increased investor confidence in the ALS drug development space.
Addressing Challenges in ALS Drug Development
ALS drug development has historically faced numerous challenges, with many promising therapies failing in clinical trials. Trace Neuroscience aims to mitigate these risks by focusing on human genetics, understanding the disease mechanism, and utilizing a proven therapeutic modality. The company's goal is to develop a treatment that can improve the lives of people living with ALS.
Green emphasized the importance of a collaborative approach, bringing together diverse expertise to translate discoveries into medicines. "One of the things I have learned over the years is that the more you can bring together a group of diverse experts who understand an area of biology, the greater chance you're going to have to be successful in translating that into a medicine," Green stated.
