Trace Neuroscience Launches Global Clinical Development Program for TRCN-1023 in ALS
Trace Neuroscience, Inc., a biopharmaceutical company focused on advancing genomic medicine for people living with neurodegenerative diseases, has announced the start of its global clinical development program for TRCN-1023, an investigational antisense oligonucleotide (ASO) designed to restore UNC13A protein function in patients with amyotrophic lateral sclerosis (ALS). The move marks a major milestone for the company as it advances a program aimed at addressing one of the most compelling genetically validated targets in ALS research.
The global clinical effort for TRCN-1023 includes the Phase 1/2 FUNCTION ALS trial, which has already received clinical trial authorization in the United Kingdom and the Netherlands, as well as LAUNCH ALS, an investigator-initiated trial currently underway in China. Together, these studies form the foundation of Trace Neuroscience’s early-stage international development strategy for TRCN-1023, a therapy the company hopes can help address the urgent unmet medical need faced by people living with ALS.
ALS is a progressive and fatal neurodegenerative disease characterized by the gradual loss of motor neurons, the nerve cells responsible for controlling voluntary muscle movement. As these neurons deteriorate, patients experience worsening weakness, loss of mobility, impaired speech and swallowing, and ultimately respiratory failure. Despite years of research and several approved therapies, treatment options remain limited, and most available drugs provide only modest benefits. Against this backdrop, the development of new targeted therapies that address underlying disease biology has become a central goal in ALS drug research.
Trace Neuroscience believes TRCN-1023 may offer a new approach by targeting UNC13A, a gene increasingly recognized as one of the most important genetic contributors to ALS pathogenesis. The company’s strategy is rooted in genomic medicine, using a precision-designed antisense oligonucleotide to influence RNA processing and restore the production of a functional protein critical to nerve and muscle communication.
A Global Clinical Strategy for an Urgent Disease
The launch of the TRCN-1023 clinical program reflects Trace Neuroscience’s intention to move quickly and build a broad early clinical data package across multiple geographies. The company’s flagship interventional study, FUNCTION ALS, is a Phase 1/2 trial that will evaluate the safety, tolerability, pharmacology, and early biological effects of TRCN-1023 in ALS patients. With regulatory clearance already secured in the U.K. and the Netherlands, the study positions Trace to begin generating clinical data in Europe as part of its first wave of human testing.
In parallel, the company is also advancing LAUNCH ALS in China, an investigator-initiated study that broadens the program’s global reach and accelerates the collection of early clinical insights. The China study is being conducted in partnership with Tenacia Biopharmaceutical, which brings neuroscience drug development and operational expertise in the Chinese market. The trial is also being led in collaboration with Yilong Wang, M.D., Ph.D., a leading neurologist at Beijing Tiantan Hospital, one of China’s premier centers for neurological care and research. According to Trace Neuroscience, the first patients in the LAUNCH ALS trial were dosed earlier this month.
By running studies across Europe and China, Trace is taking a geographically diversified approach to early clinical development. This strategy may allow the company to gather safety, biomarker, and proof-of-mechanism data more efficiently while also laying the groundwork for future international development. For a disease as aggressive as ALS, where patients and clinicians are seeking meaningful innovation as quickly as possible, the ability to generate robust early data across multiple regions could prove especially valuable.
Eric Green, M.D., Ph.D., co-founder and chief executive officer of Trace Neuroscience, said the company was founded with the goal of translating UNC13A biology into a potential medicine. He noted that Trace’s team played a role in helping establish UNC13A as one of the most compelling genetically validated targets in ALS, and described the transition of TRCN-1023 into clinical testing as a major inflection point for the company. Green added that the global early development strategy is designed to produce a strong clinical data package with the speed and urgency the ALS community needs.
Targeting UNC13A to Restore Nerve-Muscle Communication
TRCN-1023 is a highly potent and durable antisense oligonucleotide engineered to restore healthy UNC13A protein function. The therapy is administered by intrathecal injection, allowing it to reach the central nervous system directly through the cerebrospinal fluid. Once delivered, TRCN-1023 binds to UNC13A messenger RNA (mRNA) in order to regulate how the RNA is processed and to promote the formation of a functional UNC13A protein.
The scientific rationale for this approach lies in the central role UNC13A plays in synaptic transmission. The protein is essential for proper communication between nerve cells and muscle cells, helping to regulate the release of neurotransmitters at the synapse. In ALS, disruptions in UNC13A function are believed to contribute to impaired signaling at these critical junctions, ultimately affecting motor neuron performance and muscle function.
By restoring the production of functional UNC13A protein, TRCN-1023 is intended to improve communication between nerves and muscles and potentially preserve or enhance neuromuscular function. This mechanism is particularly notable because UNC13A has emerged as a target with relevance to a large proportion of ALS patients, not just a small genetically defined subgroup. That broad potential applicability could make the program especially important if the drug demonstrates convincing biological and clinical activity in early studies.
The company has described TRCN-1023 as both potent and durable, suggesting that it may be capable of producing sustained target engagement with dosing intervals appropriate for chronic neurological treatment. For ASO therapies, durability can be an important differentiator, as longer-lasting effects may help reduce treatment burden while maintaining therapeutic benefit.
UNC13A Seen as One of ALS Research’s Most Promising Targets
The launch of the clinical program comes at a time when UNC13A is drawing increasing attention across the ALS research community. Genetic and mechanistic studies have identified the gene as a key player in disease biology, and growing evidence suggests that loss of normal UNC13A function may be linked to the pathological consequences of TDP-43 dysfunction, one of the defining molecular hallmarks of ALS.
Dame Pamela Shaw, M.D., Professor of Neurology at the University of Sheffield and chief investigator of the FUNCTION ALS trial, described UNC13A as one of the most promising targets in ALS research today. She said the target is supported by strong human genetic evidence and mechanistic biology, creating a compelling rationale for therapeutic intervention. According to Shaw, restoring UNC13A function could have relevance for the vast majority of ALS patients, making it a potentially important avenue for treatment development. She added that the FUNCTION ALS trial will help build understanding of TRCN-1023’s biological and clinical effects.
The emphasis on human genetics is particularly important in the current era of neurodegenerative disease drug development. Targets backed by genetic evidence are often viewed as more attractive because they may offer a clearer connection between disease biology and therapeutic intervention. In ALS, where the underlying mechanisms are complex and heterogeneous, genetically validated targets such as UNC13A have become a focal point for precision medicine efforts.
Expanding Clinical Research in China Through LAUNCH ALS
The LAUNCH ALS investigator-initiated study in China adds another important dimension to Trace Neuroscience’s development strategy. Conducted with Tenacia Biopharmaceutical and Beijing Tiantan Hospital, the trial allows the company to extend its clinical footprint into one of the world’s largest and increasingly sophisticated drug development markets.
For ALS patients in China, the study may also represent a meaningful opportunity to access an investigational therapy based on cutting-edge genomic medicine. China has become an increasingly active participant in global neuroscience research, with expanding clinical infrastructure and growing expertise in rare and neurodegenerative disease studies. Partnering with a local organization such as Tenacia can help accelerate trial execution, navigate operational requirements, and ensure strong alignment with the local medical and regulatory environment.
Dr. Wang, principal investigator of LAUNCH ALS, said people with ALS urgently need meaningful therapeutic innovation beyond the limited options available today. He described the potency, durability, and biological rationale behind TRCN-1023 as making it a particularly exciting candidate to bring into the clinic. Wang also said he was proud to work with the Trace Neuroscience and Tenacia teams to help accelerate development of a potential new treatment for ALS patients worldwide.
A New Step in Genomic Medicine for Neurodegeneration
The initiation of the TRCN-1023 program also reflects a broader shift in the neurodegenerative disease field toward genomic and RNA-based medicines. Antisense oligonucleotides have emerged as an increasingly important therapeutic modality for neurological diseases because they can be designed to selectively target disease-associated RNA pathways and alter protein expression in ways that may not be possible with traditional small molecules or antibodies.
In ALS, this approach has already begun to shape the development landscape, particularly in genetically defined forms of the disease. Trace Neuroscience’s work with TRCN-1023 seeks to expand that paradigm by targeting a pathway with broader relevance across the ALS population. If successful, the therapy could help demonstrate how RNA-based interventions can be used not just for rare mutation carriers, but for larger groups of patients linked by common disease biology.
For Trace Neuroscience, the launch of global clinical testing marks a transition from target discovery and preclinical development into the more demanding phase of demonstrating safety, biological activity, and early efficacy signals in patients. It also positions the company among a growing group of biotechnology firms using genomic medicine approaches to tackle neurodegenerative diseases that have historically been difficult to treat.
Moving Forward in a High-Need Disease Area
ALS remains one of the most devastating conditions in neurology, with a relentless disease course and limited therapeutic options. For patients, families, and clinicians, every new program entering the clinic represents a source of cautious hope—particularly when it is grounded in a strong biological rationale and aimed at a target with broad relevance to the disease.
With FUNCTION ALS in Europe and LAUNCH ALS underway in China, Trace Neuroscience is moving forward with a development plan designed to generate early clinical insights on a global scale. The company’s decision to pursue a multi-region strategy from the outset underscores both the promise of TRCN-1023 and the urgency of bringing new ALS therapies into the clinic.
Whether TRCN-1023 ultimately succeeds will depend on what the early clinical data reveal about its safety, pharmacology, and ability to restore UNC13A function in a way that translates into meaningful patient benefit. But by launching this global program, Trace Neuroscience has taken a significant step toward testing a novel genomic medicine approach in one of the most challenging and underserved areas of neurology.
As the clinical studies progress, the ALS field will be watching closely. A therapy capable of restoring UNC13A protein function and improving nerve-muscle communication could represent an important new direction in the treatment of ALS—and potentially a broader proof point for RNA-targeted therapies in neurodegenerative disease.
About the FUNCTION ALS Phase 1/2 Clinical Trial & LAUNCH ALS IIT
FUNCTION ALS is a global Phase 1/2 randomized, double-blind, placebo-controlled clinical trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamic activity of TRCN-1023 in people living with ALS. The study is expected to enroll approximately 30 participants across sites in North America and Europe. Key eligibility criteria include age 18-75, symptom onset within the past two years or less, and slow vital capacity (SVC) of at least 60%.
Individuals with SOD1 or FUS mutations are not eligible. Participants will receive TRCN-1023 or placebo, with 24 weeks of follow-up. Designed with input from people living with ALS and their caregivers, FUNCTION ALS incorporates biomarker analyses, digital movement and speech assessments, and operational measures intended to reduce participant burden.
LAUNCH ALS is an IIT conducted in collaboration with principal investigator Dr. Yilong Wang at Beijing Tiantan Hospital to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic activity of TRCN-1023 in people with ALS. The study is expected to enroll approximately 25 participants. Eligibility criteria for enrollment are consistent with the criteria for the FUNCTION ALS trial.
About ALS
Amyotrophic lateral sclerosis (ALS, also known as motor neuron disease (MND) or Lou Gehrig’s disease), is a progressive and terminal neurodegenerative disease impacting nerve cells in the brain and spinal cord that reduces muscle function and control. As ALS advances, the ability to speak, swallow, move and breathe is increasingly impaired. In the U.S., approximately 30,000 people are living with ALS, and approximately 1 in 400 people will be diagnosed during their lifetime. Sporadic ALS that occurs without a clear family history or identified gene change is the most common form, accounting for 9 out of 10 cases, and has very limited treatment options.
About Trace Neuroscience
Trace Neuroscience is a biopharmaceutical company on a mission to expand the promise of genomic medicine for people living with neurodegenerative diseases. With an initial focus on ALS, the company is developing novel therapies to restore UNC13A protein function to re-establish healthy communication between nerves and muscle cells. Trace Neuroscience launched in 2024 with funding from leading life sciences investors and is headquartered in South San Francisco, California.
