Crucible Therapeutics develops novel therapies treating neurological diseases with significant unmet medical needs, Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal dementia (FTD). Paris, France, Manchester and Sheffield, United-Kingdom, September 21st, 2023 - Crucible Therapeutics, a biotechnology company developing novel therapies to treat amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease), the most common genetic form of motor neurone disease (MND), and frontotemporal dementia (FTD), has secured £5 million financing from Northern Gritstone and Argobio Studio. Crucible was founded in the University of Sheffield’s internationally renowned Department of Neuroscience by Professor Dame Pamela Shaw, Professor of Neurology and Director of the Sheffield Institute for Translational Neuroscience (SITraN), Professor Guillaume Hautbergue, Professor of Translational RNA Biology, and Professor Mimoun Azzouz, Chair of Translational Neuroscience, SITraN at the University of Sheffield. Research performed by the three scientific founders identified critical factors that contribute to neurotoxicity and neuronal loss in ALS and FTD. They further demonstrated, with support from LifeArc and the Motor Neurone Disease Association, and translational development support from the Cell and Gene Therapy Catapult, that one of these critical factors, SRSF1, could be targeted to improve outcomes in models of ALS and FTD. Crucible will build on its pioneering research by conducting pivotal safety and efficacy studies before proceeding with human clinical trials. Crucible is receiving financing and support from Northern Gritstone and Argobio Studio to fund essential development and manufacturing work, which will take the company up to its first clinical trial. It is the first spinout focusing on advanced therapeutics to receive investment from Northern Gritstone, the investment business focused on university spin-outs and technology-enabled businesses in the North of England. Argobio Studio is a pan-European biotech company start-up vehicle whose experienced team of entrepreneurs and scientific directors has supported multiple advanced therapeutics start-ups across Europe since inception. Demonstrating its ongoing commitment, Argobio Studio continues to offer operational guidance to Crucible throughout its spinout phase and will persist in providing support during the upcoming preclinical development stage. Neill Moray Mackenzie and Jonathan Foley from Argobio Studio will support the development of the company’s novel therapies. Together their considerable R&D, commercial and manufacturing experience in multiple gene therapy companies will be brought to bear at this critical time for Crucible. Professor Guillaume Hautbergue will join them as Non-Executive Director on the board of Crucible Therapeutics representing the founders. 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 (SITraN), said: “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. Today marks an important milestone, which brings us closer to supplying transformative treatments to people with neurological diseases like ALS (also known as motor neuron disease) and FTD. The significant experience and support that Northern Gritstone and Argobio Studio bring to Crucible further enables our pre-clinical development, and in parallel, allows us to strategically build our translational team.” Duncan Johnson, CEO of Northern Gritstone, said: “Crucible’s founders, Pamela, Guillaume and Mimoun, exemplify the world-leading scientific and medical expertise that exists in the North of England. We are delighted to be backing Crucible and we look forward to working with them as they prepare to bring the company’s therapeutic candidate closer to clinical trials. We also welcome the opportunity to invest alongside the team at Argobio Studio whose invaluable experience in gene therapy will be critical as Crucible takes the first important step in its journey.” Neill Moray Mackenzie, Entrepreneur in Residence, Partner at Argobio Studio, said: “Ever since I had tea with Stephen Hawking in Cambridge many years ago, I have been searching for a realistic hope of a cure for this devastating disease out of respect for him. At Argobio Studio, we have the opportunity to look at all the technologies with unbiased view and I can honestly say the Sheffield approach was the most compelling for us. While the therapy is still in early stages of development, we look forward to working alongside Northern Gritstone to bring the programme to fruition.”