Trace Neuroscience: slowing motor neurone disease progression with protein restoration

Problem to be solved

Motor neurone disease (amyotrophic lateral sclerosis, ALS) is a rapidly progressive neurodegenerative disorder with few effective treatment options, leading to paralysis and death typically within three to five years. Research has linked loss of the UNC13A protein to disease biology in the majority of ALS cases, creating an urgent need for therapies that can preserve or restore this pathway and slow progression.

Solution

Trace Neuroscience is developing antisense oligonucleotides (ASOs) designed to act at the genomic level to prevent the loss of UNC13A and restore healthy neuronal communication. The programme originates from research at UCL and Stanford University led by co-founders including Professor Pietro Fratta (UCL) and Professor Aaron Gitler (Stanford). While initially focused on ALS, the biology may also be relevant to related neurodegenerative conditions such as frontotemporal dementia.

How UCL Ventures helped

UCL Ventures supported Trace Neuroscience from an early stage by helping secure and manage IP protection, supporting patent filings and establishing the route to commercialisation. UCL Ventures helped put in place the exclusive licensing arrangements from UCL and supported the formation of partnerships needed to progress the ASO programme, including negotiating contracts to enable further development with experienced biotech investors and operators. 

As Dr. Caitriona O'Rourke, Senior Business Manager at UCL Ventures, says the efforts proved fruitful: says: “Third Rock Ventures (TRV), Trace and the team had in-depth discussions about the target and antisense oligonucleotide (ASO) technology and it was agreed that TRV was a fitting partner to move this ASO technology forward. UCL Ventures worked with Trace to negotiate the contracts required for the collaboration to move forward and will continue to support the academic team and Trace to move this technology to the clinic.” 

Where is Trace Neuroscience now?

Trace Neuroscience launched with a $101m Series A financing led by Third Rock Ventures, with participation from other leading life science investors. The programme is currently in preclinical development, focused on advancing ASO candidates towards first-in-human studies. Next, the company will progress translational studies, manufacturing and regulatory preparation to move the lead programme into clinical trials for ALS.