University spinout company awarded £2.3 million to develop breakthrough therapies that address underlying causes of MND

13th June 2025

Crucible Therapeutics and University of Sheffield secure a £2.3 million Innovate UK Biomedical Catalyst award to develop breakthrough siRNA therapies that address underlying causes of Motor Neuron Disease (MND).

The grant from the Innovate UK Biomedical Catalyst program will support the advancement of Crucible’s differentiated siRNA program aimed at treating MND, which is also known as amyotrophic lateral sclerosis (ALS), and other devastating neurodegenerative conditions.

The funding will enable Crucible, in partnership with Sheffield Institute of Translational Neuroscience (SITraN), to scale up manufacturing and advance its lead clinical candidate into pivotal non-clinical safety studies – an essential step toward initiating first-in-human clinical trials for MND/ALS.

Crucible’s unique therapeutic approach targets rogue RNA molecules and toxic proteins that contribute to neurodegeneration, aiming to protect motor neurons from progressive damage. This innovative strategy holds promise as a first-in-class, disease-modifying treatment for patients affected by MND/ALS.

MND/ALS is a progressive, fatal neurodegenerative disorder characterised by muscle weakness, paralysis, and ultimately respiratory failure. Toxic protein accumulation, seen in the vast majority of MND cases, directly damages motor neurons, disrupting nerve signaling and leading to severe muscle atrophy and loss of function that defines the disease.

Professor Guillaume Hautbergue, Professor of Translational RNA Biology at SITraN and Chief Scientific Officer at Crucible, said: “This grant strengthens the partnership between Crucible and SITraN, combining world-leading science with translational expertise to accelerate the development of new therapies for ALS. It exemplifies how academic–industry partnerships can transform cutting-edge research into meaningful advances for patients.”

Jonathan Foley, Executive Director and CDO of Crucible Therapeutics, said: “We are thrilled to receive Innovate UK funding as part of the Biomedical Catalyst, which will accelerate our research into a potential new treatment for ALS. This funding will support crucial translational research that brings us a step closer to delivering a therapy that could make a real difference for people living with this devastating disease.”

Duncan Johnson, CEO of Northern Gritstone, said: “Crucible’s foundations are built on the world leading expertise of Professors Guillaume Hautbergue, Mimoun Azzouz and Pamela Shaw. With support from Northern Gritstone and Argobio, Crucible is committed to developing therapies that halt the progression of neurological diseases. Winning this Innovate UK funding is testimony to the exciting potential of the science developed at the University of Sheffield”

Neill Mackenzie, Executive Chair of Crucible and EIR and Partner at Argobio, said: “This is a tremendous achievement for Crucible and the founders and scientists at the University of Sheffield. This grant puts Crucible in a strong position to develop potentially life-changing treatments for patients with devastating neurodegenerative diseases. We are very grateful to the Innovate UK Biomedical Catalyst for this recognition.”