‘Our center will tackle major challenges in gene therapy development’: University of Sheffield to house new innovation and manufacturing hub

The investment in the three ‘cutting-edge hubs’ totals £18m ($24.9m), with the sites dedicated to advancing the clinical development of new genetic treatments.

The Sheffield hub will also include a GMP (good manufacturing practice) facility that will support gene therapy projects emerging from universities across the UK. As well as manufacturing clinical grade adeno-associated viruses (AVV), it will also be ready to investigate ways to ‘radically increase yields and reduce productivity barriers in future years’.   

In addition, it will offer extensive training and skills programmes to enable the upskilling and address shortages of skills in GMP manufacturing.  

The three facilities – located in Sheffield, London and Bristol - are funded by LifeArc and the Medical Research Council (MRC), with support from the Biotechnology and Biological Sciences Research Council (BBSRC).

Gene therapy potential

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.

While the UK has a strong genetics research base, academics have found it difficult to date to get access to the clinical materials, facilities and expertise required to progress gene therapy research into clinical trials. This is where the new network comes into play.

“The gene therapy landscape at UK universities is generating world leading discoveries,” Professor Minoun Azzouz, director of the GTIMC and Chair of Translational Neuroscience at the University of Sheffield, told BioPharma-Reporter.

“However, the main barriers and challenges for translating these promising discoveries to clinical benefits and products in the markets are as follows: firstly the lack of UK GMP facilities to support clinical grade materials for UK academics; and secondly the lack of appropriate and coordinated translational support to guide the UK academics through challenging pathways to reach clinical trials and commercialisation.

“In Sheffield, the GTIMC will include a state-of-the art newly built GMP facility to supply materials with high priority to UK Universities gene therapy programmes.”

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 Sheffield GTIMC will manufacture commonly used vectors, including both lentivirus and adeno-associated virus that are needed for genetic therapy trials.

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.

In addition, it will 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.

And 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. The center will help ‘position the UK for significant bioprocessing innovation work with the potential to radically increase yields and reduce productivity barriers in future years’.

“Bioprocess innovation is a key pillar of GTIMC,” said Azzouz. “We have assembled a consortium of key players in process innovation: Cell and Gene Therapy Catapult (CGT Catapult), Centre for Process Innovation (CPI) and the Faculty of Engineering at the University of Sheffield.

“GTIMC will initially focus on adeno-associated viruses. The plan is to tech transfer an AAV from CGT Catapult to Sheffield. We will also establish new processes to increase yield and efficiency of the production systems.”

The Sheffield site will be part of a coordinated network with the two other sites - located at King’s College London and NHS Blood and Transplant in Bristol – sharing technical skills and resources to enable innovative gene therapy research.

Upskilling

To address skills shortages in the area, a new Master in Cell & Gene Therapies has been established in Sheffield.

“The course covers aspects from discovery, GMP manufacturing, process innovation, quality control, qualified person, clinical trial design, commercialisation & market authorisation,” said Azzouz.

“The rGTIMC partners – such as National Horizon Centre, CPI, CGT Catapult, Cobra, ATAC, etc – are also bringing valuable training schemes to develop highly skilled scientists and technicians in this space.”

Construction of the GTIMC building is expected in May 2022, with production to be initiated later in the year.