Our focus is on the most common genetic forms of Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) – severe, debilitating conditions for which there is currently no cure. Clearly there is an urgent medical need for new therapies.
We are pioneering RNA-targeting therapies to disrupt key disease-driving processes. Specifically, our therapies reduce toxic proteins including so-called dipeptide repeat proteins (DRP) and TDP-43 pathology.
These disease-causing proteins damage neurons, leading to progressive, life-threatening disabilities. Our approach uses a novel technology, “RNA interference” to reduce toxic protein levels, aiming to slow or preferably halt disease progression. Our goal is to develop curative treatments.
Tackling ALS – How our innovative technology works
Crucible has established a highly collaborative team of leading RNA biologists, gene therapy (GT) developers, clinical key opinion leaders, GT manufacturing specialists and successful biotech entrepreneurs.
Core to the Crucible’s approach is the development of novel therapies that prevent rogue RNAs from escaping into the cytoplasm where they cause extensive damage.
Our lead programme focuses on the neurodegenerative condition Amyotrophic Lateral Sclerosis (ALS) also known as Motor Neuron Disease (MND).
Crucible Therapeutics, a UK-based biotech company, was founded in 2023 to develop groundbreaking medicines for neurodegenerative diseases.
Our work originates from the Sheffield Institute for Translational Neuroscience (SITraN) and the Sheffield NIHR Biomedical Research Centre (BRC) at the University of Sheffield – both internationally recognized institutes in neurodegenerative disease research.
SITraN is a world-renowned institute dedicated to translating scientific discoveries into clinical treatments. Together with the Sheffield NIHR BRC, SITraN has led major clinical trials in patients with neurodegenerative disease, including Qalsody/Tofersen for SOD1-ALS and the first siRNA trial for Alzheimer’s disease. It is also home to the world’s largest ALS/MND biobank, supporting groundbreaking research in neurodegeneration.
A new approach to Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis ALS, also known as Motor Neuron Disease (MND) and Lou Gehrig’s disease is a devastating progressive and fatal neurodegenerative disease.
Crucible’s approach has the potential to prevent neurodegeneration in the majority of ALS patients (up to 97%) without impacting the normal functions of the cell.
Pioneering Treatment for Frontotemporal Dementia (FTD)
FTD is the most common form of dementia in people under the age of 60 and the second most common form of dementia overall.
Our goal is to develop a disease modifying treatment that halts neurodegeneration and enables C9ORF72-FTD patients to recover cognitive function.
Crucible Therapeutics, a biopharmaceutical company developing novel therapies for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, today announced the appointment of Dr Cliff Holloway as Chief Executive Officer. Dr Holloway will also join the company’s Board of Directors.
Crucible Therapeutics, a biotechnology company developing novel gene therapies for neurodegenerative diseases, is pleased to announce that it has been awarded a substantial £2.3M grant from Innovate UK’s Biomedical Catalyst program …
University of Sheffield spinout company, Crucible Therapeutics, a biotechnology company developing novel therapies to treat amyotrophic lateral sclerosis (ALS), and frontotemporal dementia (FTD), has secured £5 million financing from Northern Gritstone and Argobio Studio.
We seek partners with capabilities to accelerate our programs and market access, increase effectiveness, quality and productivity.
Crucible Therapeutics Limited
The Innovation Centre
217 Portobello
Sheffield S1 4DP
UK
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