Biotechnology company Crucible Therapeutics, a University of Sheffield spinout company, has been awarded £2.3m to develop breakthrough 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.
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.”