New £18m gene therapy centre will 'cure the incurable'

A new dedicated gene therapy hub for the North of England will fast-track treatments for diseases and conditions which are at present incurable.

The Gene Therapy Innovation and Manufacturing Centre (GTIMC) housed in Sheffield will be one of three cutting-edge hubs in the UK dedicated to advancing the training, capacity building and development of clinical development of new genetic treatments.

It is the only such centre north of London and will pioneer scientific discoveries into promising treatment options for millions of patients with life threatening diseases.

It is part of an £18m network created by LifeArc and the Medical Research Council (MRC), with support from the Biotechnology and Biological Sciences Research Council (BBSRC).

Professor Sherif El-Khamisy is Director of the Institute Cancer Therapeutics who led the University of Bradford in the consortium of the successful bid.

He said the scheme would develop new cures to conditions which are at present considered ‘incurable’, including inherited disorders.

Prof El-Khamisy said: “Gene therapy has huge potential. It has already led to cures for conditions such as spinal muscular atrophy (SMA). In the future, we will be looking at conditions like motor neurone disease, Alzheimer’s and rare cancers. 

“This new centre also means we will have capacity to convert fundamental discoveries into actual products, instead of giving them away to the US.”

He added: “This is significant because it will be the only hub in the whole of North, the only place we can get student training, capacity building and manufacture, which will then act as a catalyst for grant applications in the future.

“Gene therapy will be subject to strict regulatory approvals and clinical trials. In short, there are disorders which cannot be cured by traditional therapeutics, including SMA and MND - gene therapy is expected to deliver cures for those disorders.”

Gene therapy is a promising treatment option for more than 7,000 rare diseases that currently have no cure. It aims to treat these conditions, by engineering another gene to replace, silence or manipulate the faulty one.

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.

Professor Mimoun Azzouz, Director of the GTIMC and Chair of Translational Neuroscience at the University of Sheffield, said: “The Gene Therapy Innovation and Manufacturing Centre will tackle major challenges in gene therapy development for some of the most devastating diseases.

“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.”

He added: “This is a momentous milestone for revolutionary medical advances not only for Sheffield and South Yorkshire, but also for the UK.”

Professor Dame Pam Shaw, Director of the NIHR Sheffield Biomedical Research Centre and co-applicant on the GTIMC application said: “This exciting development and partnership will speed up the pull through of new gene therapies into early phase clinical trials and offer hope to patients with neurological and other rare diseases that can be addressed in this way.
“The support given to this initiative will greatly accelerate the translational potential of genetic therapies in the UK and bring benefits in key areas of unmet medical need.”

Professor Fiona Watt, MRC's Executive Chair, said: “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.”

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 academicled 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.

Additional information

Alongside the national network funding from LifeArc, the MRC and BBSRC, the GTIMC was made possible thanks to a £3m donation from The Law Family Charitable Foundation, established by Andrew Law and his wife Zoë. This funding was part of a record £5m donation from the University of Sheffield alumnus, which will also see the launch of a new student support programme.

The GTIMC is planned for a site on the University of Sheffield’s Innovation District close to existing translational research facilities and will contribute to an ongoing programme of regional investment and regeneration.

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, Kings 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