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Trace Neuroscience launches with exclusive license from UCL and $101 million Series A funding to take forward potential new therapeutic for motor neurone disease

12 November 2024

Conceptual illustration of neuron cells.

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One of the largest biopharma Series A rounds in recent years will fund development of a treatment for amyotrophic lateral sclerosis (ALS), which affects 5,000 people in the UK

Today sees the launch of Trace Neuroscience, Inc., a biopharmaceutical company whose co-founders include Pietro Fratta, MD, PhD, Professor of Cellular and Molecular Neuroscience at University College London (UCL) and Aaron Gitler, PhD, Professor of Genetics at Stanford University. Trace Neuroscience licensed intellectual property exclusively from UCL as part of the company formation.

Trace Neuroscience is developing antisense oligonucleotides – nucleic acids that can act at the genomic level to restore the production of a key protein called UNC13A, an essential component for neuronal communication in the brain and spinal cord. The decline in people’s ability to produce UNC13A characterises almost all cases of ALS, also known as motor neuron disease, a rapidly progressive neurodegenerative disorder leading to wasting, paralysis, and eventual death from respiratory failure within 3 to 5 years. By restoring UNC13A protein levels, healthy communication between nerves and muscle cells impacted by neurodegenerative disease can be re-established thereby slowing the progression of the disease.

The co-founders of Trace Neuroscience are leaders in genomic medicine innovation. Their simultaneous research into the role of UNC13A in ALS led to their co-founding of the company to bring a treatment to market to benefit patients, who currently have few treatment options.

Professor Pietro Fratta, based at UCL Queen Square Institute of Neurology, said: “UNC13A is critical for neurons to communicate amongst each other and with muscles, and is lost in nearly all ALS cases. Being able to re-establish this is groundbreaking. This large funding round is pivotal for us to rapidly translate this science into a life-changing medicine and advance our lead program toward the clinic.”

The $101 million financing was led by Third Rock Ventures and included participation from Atlas Venture, GV and RA Capital Management.

UCL Business (UCLB) – the commercialisation company for UCL – supported the academic team working closely to protect the intellectual property and with the Third Rock and Trace teams facilitating the collaboration with Pietro and his team at UCL.

Caitriona O’Rourke, Business Manager at UCL Business (UCLB), said: “This Series A funding round indicates investors’ excitement for UNC13A as a therapeutic target and commercial applications for this novel therapeutic. We share this excitement and are proud to support Professor Fratta and the entire Trace Neuroscience team as they move this candidate from the lab into the clinic and hopefully to the market.”

Targeted Approach, Broad Impact, Exciting Potential

Approximately 97% of people with ALS produce insufficient amounts of UNC13A, which in turn is regulated by a protein called TDP-43 that controls RNA splicing. When TDP-43 stops functioning normally, the UNC13A messenger RNA (mRNA) is improperly spliced and this hinders production of UNC13A, which is vital for correct neuron signalling from brain and spinal cord to muscles.

“Trace was founded on the breakthrough discovery that loss of UNC13A is a driver of disease in nearly all people with ALS,” said Eric Green M.D., Ph.D., co-founder and CEO of Trace Neuroscience. “We have now brought together academic leaders, experienced drug developers and a leading syndicate of investors committed to urgently turning this discovery into a medicine. I look forward to continuing our fruitful collaboration with Pietro and his team at UCL on this journey.”

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Media Contact: Nigel Campbell, UCL Business Ltd:  nigel.campbell@uclb.com

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 Third Rock Ventures and other leading life sciences investors, and is headquartered in South San Francisco, CA. For more information, please visit us at www.traceneuro.com and follow us on LinkedIn and X.

About UCL Business

UCLB is part of UCL, one of the world’s leading universities. We make sure the exceptional ideas and innovations generated by researchers at UCL and its associated hospitals – University College London Hospitals, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital – have real world impact, creating benefits for society and bringing revenue back to the university. UCLB’s track record of success includes over £2.85 billion raised in investment for UCL spinouts, bringing pioneering technologies from the laboratory to market. For more information visit www.uclb.com