Crucible Therapeutics spins out with £5m

University of Sheffield spinout gets £5m for gene therapies targeting ALS and FTD, advancing towards clinical trials.

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Crucible Therapeutics Ltd., the newly launched spinout company from the University of Sheffield, secured the investment of £5m from Northern Gritstone Ltd. and Argobio Studio SAS on September 21st. The company, is developing novel gene therapies to treat severe neurological conditions, including amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease, the genetic form of motor neurone disease (MND), and frontotemporal dementia (FTD) cases with the C9orf72 gene mutation.

Research performed by the scientific founders has identified critical factors that contribute to neurotoxicity and neuronal loss in ALS and FTD, and one of these critical factors, SRSF1, could be targeted to improve outcomes in models of ALS and FTD. This recent funding will support pivotal safety and efficacy studies, a significant step toward human clinical trials. “Our novel therapeutic approach has made significant strides in our pursuit of transforming how these devastating diseases are treated and with the potential to achieve meaningful therapeutic impact”, said Professor Dame Pamela Shaw, Co-Founder of Crucible Therapeutics, Professor of Neurology at the University of Sheffield, and Director of the Sheffield Institute for Translational Neuroscience.

This investment not only bolsters Crucible’s pioneering work but also underscores the collaborative power of scientific innovation and partnerships in the quest to combat debilitating neurological diseases. With Northern Gritstone and Argobio Studio’s support, Crucible is poised to accelerate its therapies, offering renewed hope to those affected by ALS, MND, and FTD.

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