UCL scientists receive £78 million funding boost to develop treatment for motor neurone disease

A funding boost of more than £78 million will help UCL scientists working to develop a potential treatment for motor neurone disease, for which there is currently no cure and which affects 5,000 people each year in the UK.

Trace Neuroscience, a UCL spinout biopharmaceutical company, is working on developing a way to restore the production of a protein that is essential for communication in the brain and spinal cord.

Amyotrophic lateral sclerosis (also known as ALS) is the most common motor neuron disease; it affects the brain and spinal cord by attacking the neurons and nerves which control movement, causing them to die.

Trace Neuroscience was founded on the simultaneous "breakthrough discovery" at UCL and Stanford University that the loss of a protein called UNC13A is a driver of disease progression in nearly all people with motor neurone disease, leading to wasting, paralysis and eventual death from respiratory failure within three to five years.

Trace Neuroscience co-founder Professor Pietro Fratta (UCL Queen Square Motor Neuron Disease Centre, UCL Queen Square Institute of Neurology) found that by restoring UNC13A protein levels, healthy communication between nerves cells impacted by neurodegenerative disease could be re-established, thereby potentially slowing the progression of the disease.

Professor Fratta 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 programme towards the clinic."

Trace Neuroscience was founded on the breakthrough discovery that loss of UNC13A is a driver of disease in nearly all people with ALS.

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."

Eric Green, co-founder and CEO of Trace Neuroscience

Trace Neuroscience licensed intellectual property exclusively from UCL as part of the company formation, and the $101 million USD 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 Professor Fratta and his team at UCL.