NEW YORK – Crucible Therapeutics, a spinout of the University of Sheffield in England, has raised £5 million ($6.1 million) to support development of gene therapies for certain neurodegenerative diseases linked with mutations of the C9orf72 gene.
The £5 million in financing from investment firm Northern Gritstone and European startup studio Argobio Studio will fund preclinical development and manufacturing work that aims to bring Crucible Therapeutics through to its first clinical trial, University of Sheffield said Monday. The company is developing therapies for motor neuron disease (MND), amyotrophic lateral sclerosis (ALS), and frontotemporal dementia (FTD).
Crucible Therapeutics, which launched this year, was founded on research from three professors at the University of Sheffield's neuroscience institute: Guillaume Hautbergue, a professor of translational RNA biology; Mimoun Azzouz, chair of translational neuroscience; and Dame Pamela Shaw, director of the Sheffield Institute for Translational Neuroscience. The trio found that reducing or altering the makeup of a protein, SRSF1, could potentially treat neurodegenerative conditions by reducing the amount of pathological RNA molecules that escape into the cell's cytoplasm.
"Our novel therapeutic approach has made significant strides in our pursuit of transforming how these devastating diseases are treated," Shaw, one of the company's cofounders, said in a statement. "Today marks an important milestone, which brings us closer to supplying transformative treatments to people with neurological diseases like MND and FTD."