Three new pioneering research hubs are being established to advance scientific discoveries into promising treatment options for millions of patients with life-threatening diseases.
The research facilities will help to form a national network of cutting-edge gene therapy innovation hubs, backed by £18million in funding to support the clinical development of new genetic treatments.
The innovation hubs – funded by LifeArc and the Medical Research Council (MRC), with support from the Biotechnology and Biological Sciences Research Council – have been hailed as bringing fresh hope of breakthroughs for people living with one of the more than 7,000 genetic diseases that currently have no cure.
Neurological conditions are a key part of the research, with Alzheimer’s disease, Motor Neurone Disease, Multiple Sclerosis, Huntington’s disease and Parkinson’s disease being among those which are currently incurable.
Gene therapy aims to treat these conditions, among with the thousands others, by engineering another gene to replace, silence or manipulate the faulty one.
The three national hubs will be the University of Sheffield Gene Therapy Innovation Manufacturing Centre (GTIMC); the NHS Blood and Transplant Gene Therapy Hub in Bristol; and The King’s College London/Royal Free/UCL Gene Therapy Hub.
They will operate as a coordinated network, sharing technical skills and resources to enable innovative gene therapy research, and will enable academic-led clinical trials of novel gene therapies to take place, helping the most innovative research to reach patients.
“Recent innovations in gene therapies hold enormous potential for treating conditions such as rare diseases, but often promising ideas – particularly in academia – are not making it through to patients,” says Dr Melanie Lee, CEO of LifeArc.
“Through our collaboration, we aim to meet the need for researchers to have access to the essential facilities and translational advice to progress promising research.”
“The new network of Innovation Hubs for gene therapies will build on the UK’s great strengths in this area, providing targeted investment in vital infrastructure to accelerate academic research programmes down the path to patient benefit, supporting the delivery of a new wave of genetic medicines,” continues Professor Fiona Watt, MRC’s Executive Chair.
Sheffield’s GTIMC, led by Professor Mimoun Azzouz, builds on a strong history of translational gene therapy research at the University of Sheffield, working with partners.
The centre includes a new state-of-the-art modular GMP manufacturing facility, located at the University of Sheffield’s Innovation District. It will support gene therapy projects emerging from UK universities.
“The Gene Therapy Innovation and Manufacturing Centre will tackle major challenges in gene therapy development for some of the most devastating diseases,” says Professor Azzouz.
“Gene therapies are pioneering medical advances that have the potential to offer much needed, novel, effective treatments for many rare and incurable diseases that cannot be treated by conventional drug compounds.
“Sheffield has emerged as one of the leading players in the cell and gene therapy and this national network of partners, facilities and training programmes will allow us to keep pace with translational discoveries for new and potentially life changing treatments.
“This is a momentous milestone for revolutionary medical advances.”
The NHSBT Gene Therapy Hub will be hosted within a new 1,000sq m facility for the production of gene therapies under GMP.
It will be funded jointly by NHSBT and the Department of Health and Social Care and under construction at the NHSBT Filton Blood Centre (Bristol).
Due to be operational by the end of 2021, the new hub will support early phase academic-led gene therapy trials and facilitate the provision of cost effective viral vectors and plasmid DNA to stimulate the UK’s gene therapy sector.
The King’s College London/Royal Free/UCL Hub will provide a comprehensive capability for clinical grade viral vector manufacturing.
This will include both AAV and lentivirus production for early phase trials, alongside substantial programmes in process innovation, knowledge transfer, and training to address critical skills shortages.
