New gene therapy innovation centre to advance scientific discoveries into life-changing treatments

A pioneering gene therapy innovation centre at the University of Sheffield is set to advance scientific discoveries into promising treatment options for millions of patients with life-threatening diseases.

The Gene Therapy Innovation and Manufacturing Centre (GTIMC), led by Professor Mimoun Azzouz, is one of three pioneering hubs announced today (18 March 2021) in a new £18 million network funded by LifeArc and the Medical Research Council (MRC), with support from the Biotechnology and Biological Sciences Research Council (BBSRC).

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.

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

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”

The new centre, which will be the first in the north of England, builds on the University of Sheffield’s strong history of translational research and its reputation as an international centre of excellence for gene therapeutics.

Dr Lucy Foley, Director of Biologics at CPI, said: ​“CPI is delighted to partner with the new Gene Therapy Innovation and Manufacturing Centre to support the development, scale up and commercialisation of viral vector based gene therapy products, by providing access to expertise and facilities to enable these life changing therapeutics to progress to the clinic and future commercial manufacture.”

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.”

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.

Professor Koen Lamberts, President and Vice-Chancellor of the University of Sheffield, said: ​“At the University of Sheffield we focus our research on finding real-world solutions to some of the biggest global challenges. The Gene Therapy Innovation and Manufacturing Centre will unlock development pathways for new treatments for people affected by devastating genetic disorders, many of which have no cure.

“We are delighted that our University is at the forefront of research in this pioneering field of medicine and that this new centre will build on our reputation as an international centre of excellence for gene therapeutics.

“This is fantastic news for the City Region and the North of England and we look forward to working collaboratively to share technical skills and resources across the coordinated network.”

Alongside the national network funding from LifeArc, the MRC and BBSRC, 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 million donation from the University of Sheffield alumnus, which will also see the launch of a new student support programme.

Andrew Law, who is 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.”

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.

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.”