The construction of a pioneering gene therapy innovation centre in Sheffield is now complete. The centre is set to advance scientific discoveries into promising treatment options for millions of patients with life-threatening diseases.
Gene therapy is a promising treatment option for more than 7,000 rare diseases that are currently without a cure. The new University of Sheffield Gene Therapy Innovation and Manufacturing Centre (GTIMC) aims to treat these conditions by engineering another gene to replace, silence or manipulate the faulty one.
The state-of-the-art centre will bring together academic institutions, NHS trusts, non-profit and industry partners across the north of England, Midlands and Wales enabling academic-led clinical trials of novel gene therapies. The GTIMC will deliver essential translational and regulatory support alongside extensive training and skills programmes to enable upskilling and address shortage of skills in Good Manufacturing Practice (GMP) manufacturing.
The GTIMC is one of three pioneering hubs in a new £18 million network funded by LifeArc and the Medical Research Council (MRC), with support from the Biotechnology Sciences Research Council (BBSRC).
Professor Mimoun Azzouz, director of the GTIMC and chair of Translational Neuroscience at the University of Sheffield, said: “Sheffield is internationally renowned for its world-class rare diseases research. This centre will help to accelerate this revolutionary research into promising and innovative treatment options for many rare diseases which currently have no cure.
“Sheffield has emerged as one of the leading players in cell and gene therapy and this national network of partners, facilities and training programmes will allow us to stay at the cutting edge of translational discoveries for new and potentially life changing treatments.
“Seeing the construction work completed is an exciting milestone for the team. It brings us closer to being fully operational and able to progress new and exciting discoveries which will benefit patients and families worldwide. Our focus now is to secure MHRA GMP licensing.”
Alongside the national network funding from LifeArc, the MRC and BBSRC, as well as support from the South Yorkshire Mayoral Combined Authority, the GTIMC was made possible thanks to a £3 million donation from The Law Family Charitable Foundation, established by Andrew Law and his wife Zoë. This funding was part of a record £5.85 million donation from the University of Sheffield alumnus, which will also see the launch of a new student support programme.
Andrew Law, chairman and CEO of Caxton Associates, said: “The University of Sheffield is rapidly developing a global reputation in gene therapy. The new Gene Therapy Innovation and Manufacturing Centre will drive innovation and world-class research, while presenting a real opportunity to catalyse the creation of new start-up companies to facilitate commercialisation in the North.
“This investment will enhance vital partnerships with biotechnology and pharmaceutical companies to help accelerate gene therapy programmes and clinical trials, at the same time as supporting regional economic growth and job creation.”
It is expected the GTIMC will be open and fully operational in early 2023. The centre will include a cutting-edge GMP (good manufacturing practice) facility that will support gene therapy projects emerging from universities across the UK.
The facility will utilise highly efficient processes to manufacture clinical grade adeno-associated viruses (AAV) and provide all the necessary quality assurance, regulatory certification and governance for human trials at Advanced Therapies Treatment Centres and NHS trusts within the GTIMC and the national network.
The three national hubs, located at the University of Sheffield, King’s College London, and NHS Blood and Transplant in Bristol will operate as a coordinated network, sharing technical skills and resources to enable innovative gene therapy research.
Dr Melanie Lee, CEO of LifeArc, said: “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. 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 GTIMC will manufacture commonly used vectors including both lentivirus and adeno-associated virus that are needed for genetic therapy trials, while positioning the UK for significant bioprocessing innovation work with the potential to radically increase yields and reduce productivity barriers in future years. The hub network will also design and share commercially ready platforms, using common cell-lines, plasmids and reagents to reduce costs, facilitate simplified licensing agreements and streamline regulatory reviews. A key aim is to smooth the transition between small-scale supply for early clinical trials through to larger-scale manufacture for patient trials, and beyond.
Professor John Iredale, MRC’s interim executive chair, said: “The 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.”