LifeArc and the Medical Research Council (MRC), with support from the Biotechnology and Biological Sciences Research Council (BBSRC), have announced £18m investment to establish a network of Gene Therapy Innovation Hubs.
The funding supports the creation of three new dedicated facilities to enable more rapid development of novel gene therapies to take place in the UK. By providing materials and support for academic-led clinical trials, the Hubs have the potential to accelerate treatment for millions of patients including those with rare and life-threatening genetic diseases.
The new Hubs will unlock development pathways for novel genetic medicines by offering access to GMP (good manufacturing practice) facilities for clinical trials materials, alongside essential translational support and regulatory advice.
The Hubs will be at Kings College London, NHS Blood and Transplant in Bristol, and the University of Sheffield. These centres will operate as a coordinated network, sharing technical skills and resources to help UK academics to progress their novel gene therapy research into clinical trials.
The Hub network will:
- Manufacture GMP-grade of the viral vectors, lentivirus (LV) and adeno-associated virus (AAV), for academic-led early phase clinical trials
- Support the translation of gene therapy projects from the academic lab into early clinical trials.
- Deliver an extensive training and skills programme to generate a highly skilled workforce to address the shortage of skills in GMP manufacturing
- Undertake programmes of process development and innovation to enhance manufacturing efficiencies
- Design and share commercially ready platforms, using common cell-lines, plasmids and reagents to facilitate the transition between small-scale supply for early clinical trials through to larger-scale manufacture for patient trials, and beyond
About the centres
The University of Sheffield Gene Therapy Manufacturing Centre (GTIMC)
The GMTIMC includes a new, state-of-the-art modular GMP manufacturing facility, located at the University of Sheffield’s Innovation District. The facility will support gene therapy projects emerging from UK universities and will generate clinical-grade AAVs along with the necessary quality assurance, regulatory certification and governance.
NHS Blood and Transplant Gene Therapy Hub
The NHSBT Gene Therapy Hub will be hosted within a new, state-of-the-art facility that will produce gene therapies under GMP. The facility is currently under construction at the NHSBT Filton Blood Centre in Bristol. Due to be operational by the end of 2021, it will support early phase academic-led gene therapy trials and facilitate the provision of cost-effective viral vectors and plasmid DNA to stimulate the UK’s gene therapy sector.
The King’s College London/Royal Free/UCL Gene Therapy Hub
The King’s College London/Royal Free/UCL Gene Therapy Hub will provide a comprehensive capability for clinical-grade viral vector manufacturing. This will include both AAV and lentivirus production for early-phase trials, alongside substantial programmes in process innovation, knowledge transfer, and training to address critical skills shortages. As part of the network, the centre will ensure that the UK capitalises on its outstanding academic medical research to deliver novel gene therapies to patients, providing transformative treatments for currently intractable conditions.
What are the challenges faced by UK academics working in gene therapy research?
UK academics are struggling to access clinical-grade viral vectors and the translational science expertise to help advance their research towards early patient trials. As a result, many promising translational research programmes face lengthy delays.
How will the Hubs support academics to progress gene therapy research?
Through the Hub network, academic researchers will share resources, knowledge and capabilities in translating promising gene therapy research. This will accelerate the progression of research discoveries towards clinical trials and commercial development so that patients can access new treatments faster.
How will the Hubs be set up and monitored?
A Coordinating Committee will oversee the creation and ongoing operation of the Hub network to promote sharing of knowledge and capabilities, engage with the academic community and foster interactions with commercial organisations to facilitate the onward development of new genetic medicines.
What will happen once the 5-year funding period expires?
The Hubs will use the current funding to establish sustainable facilities, including vital equipment, platforms and expertise, ideally positioned to support the UK academic landscape in the long term.
How many academic trials will the Hubs be able to support?
Each of the Hubs will be able to support between three and five projects per annum when they are running at capacity.
How can academic researchers access the Hubs?
All academic projects will be able to apply for access to the Hubs. The Coordinating Committee will have responsibility for assessing and allocating projects across the network. Further information on how to access the hubs will be made available in the second half of 2021 when potential projects can be submitted for discussion with the network. Initial enquiries can be made via email@example.com
What are the eligibility criteria for projects?
The primary purpose of the Hubs is to serve UK-based academic research requiring access to GMP-grade viral vectors. Projects being submitted for access will require funding to cover their individual running costs. However, the hubs will be able to assist in developing funding applications.
Projects will be prioritised as follows:
- MRC and/or LifeArc funded academic projects
- UK-based, third party funded academic projects
- UK-based, early-stage (seed) academic-led (i.e., PI founder involvement) projects
- Other (including commercial projects where there is spare capacity)
Is this just for UK academics?
The primary purpose of the Hubs is to serve the UK academic community which has been under-served with access to GMP manufacturing. It is expected that, at least in the short-term, this will take up the capacity of the Hubs.
For enquiries about the Gene Therapy Innovation Hubs, please get in touch with firstname.lastname@example.org