New treatment for Motor Neurone Disease to be trialled

A pioneering experimental treatment for Motor Neurone Disease, developed by UCL spinout Trace Neuroscience, will be trialled in humans as part of a new international clinical programme.

The biopharma company, co-founded by Professor Pietro Fratta (UCL Queen Square Institute of Neurology) with the support of UCL Ventures, has opened two trials for the drug TRCN-1023, a potential new treatment for amyotrophic lateral sclerosis (ALS) – the disease’s most common subtype of Motor Neurone Disease. 

MND/ALS is a devasting and ultimately fatal disease, that causes progressive muscle weakness due to the degeneration of motor neurons in the brain and spinal cord. As the disease advances, patients gradually lose the ability to move, speak, swallow and breathe. It is estimated to affect 1 in 300 people in their lifetime, and there are currently no disease-modifying drugs available to treat the condition.

The candidate therapeutic is designed to target one of the most important genetic drivers of MND/ALS, a protein called UNC13A. Loss of UNC13A function is believed to contribute to disease progression in around 97% of people with MND/ALS, making it a key target for potential therapies. It is hoped that TRCN-1023 will help restore the expression of the gene which results in UNC13A being produced in nerve cells.

Professor Fratta’s research at UCL helped establish the biological importance of the UNC13A protein in causing MND/ALS. 

The clinical trial is called FUNCTION ALS – a Phase 1/2 clinical trial authorised to begin in September in the UK, with plans to expand to other countries in 2026. It follows an initial trial (LAUNCH ALS) which took place in China, where the first patients have recently been dosed. Together, these trials are designed to accelerate development and build early global clinical evidence for TRCN-1023. 

How the drug TRCN-1023 works

While sometimes grouped alongside “genetic medicines,” it is not a gene therapy in the traditional sense (it does not permanently alter DNA). Instead, it works at the level of RNA - the intermediary between genes and proteins.

A short, synthetic strands of nucleic acid, TRCN-1023 is designed bind to RNA inside cells to correct the processing of UNC13A RNA, enabling the body to produce a functional version of the protein.

Professor Fratta said: “UNC13A is critical for neurons to communicate amongst each other and with muscles and is lost in nearly all MND/ALS cases.

“Our research at UCL helped uncover just how central this protein is to the disease process, making it one of the most compelling and widely relevant targets for new treatments.

“Seeing this discovery translated into a clinical trial is a major milestone. It brings us a step closer to a therapy that could make a meaningful difference for the vast majority of people living with MND/ALS.”

Eric Green, Co-founder and CEO of Trace Neuroscience “Our team helped establish UNC13A as one of the most compelling genetically validated targets in ALS, and we built Trace Neuroscience to translate that biology into a medicine.

“We’re now excited to advance TRCN-1023 into the clinic through a global strategy designed to generate robust data with the urgency this disease demands.”

Trace Neuroscience: a UCL spinout

UCL Venture worked closely with the academic team to establish Trace Neuroscience, protect the intellectual property, and with investors facilitate the collaboration with Professor Fratta and his team at UCL.

Rick Fagan, Director of Biopharm at UCL Ventures, said:

 “When Trace Neuroscience launched in 2024, they did so with a milestone Series A funding round of over $100m, indicating investors’ excitement for UNC13A as a therapeutic target. 

“We share that excitement and are proud to support Professor Fratta and the entire Trace Neuroscience team as this therapeutic now moves into clinical trials. That’s what technology transfer is all about – getting world-leading research from the university out into the world where they have the potential for bringing positive impacts.”

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