Innovation Hubs for Gene Therapy: A national network to transform gene therapy research
Our partnership with the Medical Research Council (MRC) and the Biotechnology and Biological Sciences Research Council (BBSRC) has created an £18 million network of gene therapy hubs to advance promising research into new treatments for patients.
Overview
Three dedicated facilities will advance the clinical development of new genetic treatments, with potential to transform care for millions of patients including those with rare and life-threatening genetic diseases. The Hubs will be at King’s College London, NHS Blood and Transplant in Bristol and the University of Sheffield.
About the Innovation Hubs for Gene Therapy
The Innovation Hubs aim to transform the development of gene therapies by addressing the challenges of manufacturing, regulatory compliance, and translational expertise. They provide researchers with access to cutting-edge facilities and expert support to overcome barriers in translating academic gene therapy research into clinical trials.
The Hubs are equipped to manufacture essential viral vectors while developing scalable and efficient production methods. By leveraging shared resources and expertise across the network, they hope to lower costs, streamline licensing processes, and expedite regulatory approvals.
he Hubs are also addressing workforce challenges by implementing comprehensive training programs to bridge the skills gap in gene therapy manufacturing. In 2023, they published a report addressing the skills gap in the UK cell and gene therapy sector.
The University of Sheffield Gene Therapy Innovation Manufacturing Centre (GTIMC)
Led by Professor Mimoun Azzouz, the GTIMC leverages Sheffield’s expertise in gene therapy research. Her Royal Highness The Princess Royal officially opened the facilities in October 2024. It is located in Sheffield’s Innovation District and has the capabilities to produce clinical-grade AAVs for trials, supported by comprehensive quality assurance and regulatory certification.
In addition to manufacturing, the GTIMC provides training programmes to upskill researchers and address the shortage of expertise in Good Manufacturing Practice (GMP) production.
NHS Blood and Transplant (NHSBT) Gene Therapy Hub
Located in Bristol, the NHSBT Hub produce GMP-grade viral vectors, including AAV and lentivirus, and plasmid DNA for early-phase trials. Led by Dr Jon Smythe and Dr Paul Lloyd-Evans, this facility supports academic research teams in translating their findings into clinical applications.
The hub also delivers training programmes and collaborates with other hubs to develop a skilled workforce, ensuring the UK remains competitive in the gene therapy sector.
The King’s College London/Royal Free/UCL Gene Therapy Hub
Led by Professor Robin Ali, this hub specialises in manufacturing GMP-grade AAV and lentivirus vectors for early-phase clinical trials. It will also drive innovation in production processes and provide training to address critical skill gaps in the field.
Ultimately, the hub aims to deliver novel treatments for previously untreatable conditions by harnessing the UK’s strengths in academic research, creating significant patient and economic benefits.
About gene therapy
Gene therapies have the potential to deliver treatments for a wide range of conditions, including around 7000 rare diseases (the majority of which are caused by one faulty gene). Gene therapy aims to treat – or even cure – these conditions, by engineering another gene to replace, silence or manipulate the faulty one. The therapeutic gene is frequently introduced via a ‘viral vector’, a virus particle modified to remove all unwanted or harmful properties.
Gene therapies offer huge potential as treatments for a wide range of conditions and the UK has a world-class genetics research base – however, to date, academics have found it difficult to get access to the clinical materials, facilities and expertise required to progress gene therapy research into clinical trials.
The Innovation Hubs address these barriers, enabling efficient production and testing of therapies to bring life-changing treatments to patients more rapidly.
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