University of Sheffield and CGT Catapult Launch Innovative Gene Therapy Program

The University of Sheffield, in collaboration with the Cell and Gene Therapy Catapult (CGT Catapult), has launched an innovative gene therapy program that could pave the way for novel treatments for neurodegenerative diseases.

LifeArc and the Motor Neurone Disease Association have awarded £513,141 (US$715,902) to the research so that the gene program can be tested for feasibility and efficiency to treat motor neurone disease (MND) and frontotemporal dementia (FTD) patients with underlying mutations in a causative gene known as C9orf72.

The research is being led by Guillaume Hautbergue, head of the RNA Biology Laboratory, in collaboration with Mimoun Azzouz and Dame Pamela Shaw, all from the University of Sheffield’s Institute for Translational Neuroscience. Additionally, the work will be conducted in collaboration with the CGT Catapult, which will provide a detailed non-clinical safety strategy and regulatory advice to guide the team from the University of Sheffield for the potential commercialization of an advanced therapy medicinal product.

“This is a completely new approach to tackling the most common type of [MND],” said Hautbergue, in a July 29, 2021 press release. “No one has yet attempted to prevent these repeated sequences of RNA from leaving the cell’s nucleus and it opens up new areas of investigation for gene therapy. It is an exciting time for biomedical research and the development of novel treatments for incurable diseases such as MND.”

“The progress of this scientific breakthrough from the University of Sheffield and the support for this program by leading investors and charities such as LifeArc and the MND Association, is testament to the UK research excellence and favorable ecosystem for cell and gene therapies,” commented Matthew Durdy, CEO, CGT Catapult, in the press release. “We welcome the opportunity to be part of this program and look forward to working with the University of Sheffield to help advance their innovative gene therapy technology into the clinics.”

Source: CGT Catapult