NHSBT Innovation Hub for Gene Therapies

Many diseases, including cancer, heart disease and diabetes, are caused by the body's cells and tissues malfunctioning. The behaviour of any cell is 'programmed' by its genes and researchers have found that cell behaviour can be altered by changing DNA, RNA and other genetic material in the cell. Gene therapies work by inserting new genetic material into malfunctioning cells, and 'reprogramming' them to function more normally, or by inserting genetic material into a normal cell to produce a new protein (e.g. for a vaccine). Gene therapy offers hope to patients with diseases that have been, up to now, incurable. The UK has excellent teams of researchers who are exploring this exciting topic.

Unfortunately, some of the essential materials for gene research are in short supply in the UK, as are skilled technicians to work on the research. There is a particular shortage of viral vectors - special viruses that can be 'loaded' with genetic material and inserted into malfunctioning cells, where their 'payload' replaces genes causing the malfunction. The UK's scientists are held back by these shortages and forced to rely on overseas suppliers; or research is delayed due to difficulties in recruiting staff. This leads to delays in the discovery of new treatments for desperately ill patients.

Another important challenge for UK researchers is that achieving great results in the lab is only half the story - before a new treatment can be offered to patients, it must be approved: shown to be safe, effective and affordable. These new treatments are great opportunities for new companies, new jobs and for the UK economy, so academics need support in patenting their discoveries, in undertaking clinical trials and in setting up companies to make new therapies.

The MRC and LifeArc have recognised these important issues and are funding a network of hubs around the UK to generate the vital components for gene therapies and to train the skilled personnel needed. The NHSBT Innovation Hub for Gene Therapies will link with the other new hubs around the UK to address these needs. We will work with academic teams to produce the gene therapy components needed, we will train technicians and scientists in producing these at the quality needed for use in clinical trials, and we will support academics in taking their results from the lab to the hospital and the market.

Our plan is as follows:
1. To agree our role in the network with the other hubs, MRC, LifeArc and Cell and Gene Therapy Catapult (C>C). We are particularly well placed for flexible viral vector production, quality control and training, and we expect to contribute this to the network, while also collaborating with C>C on production, with LifeArc on commercialisation and patenting, and with other hubs, as requested by the network coordinators..
2. To install a new viral vector production platform, already selected by C>C as being particularly suitable to meet the needs of UK academic teams. We'll then work with the C>C to ensure it's certified for quality assurance (and so ready to produce clinical grade components). This new platform will produce a specific type of viral vector - adeno-associated-viral vectors (AAVs); this will complement our other platform, producing lentiviral vectors, as well as our existing platform for plasmids (another important gene therapy ingredient).
3. To be assigned academic clients and their projects by the coordinating committee, and then to work with these teams to put in place a production line and training programme that meets their needs. A flexible and customised approach is important, as every academic project will be different.
4. To build a reputation and customer base that makes the NHSBT Hub a 'go to' destination for viral vector services. As a not-for-profit NHS organisation, our aim is to be sustainable, provide excellent value for the benefit of the UK gene therapy community and, ultimately, to save and improve more lives.

The core ambition for the NHSBT Innovation Hub for Gene Therapies (the NHSBT Gene Therapy Hub), will be to 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.

Our Hub will provide viral vector manufacturing, training and support services for academic-led groups seeking Adeno Associated Viral (AAV) vectors at GMP and research-grade qualities.

The NHSBT Gene Therapy Hub will be hosted within the newly-expanded NHSBT Clinical Biotechnology Centre (CBC), at NHSBT Filton, scheduled to be fully operational by end 2021. The CBC has supported UK academics through the manufacture of plasmid DNA and provision of GMP, quality and other services for more than 15 years. The expanded CBC is an ideal environment for the NHSBT Hub, with an experienced and multidisciplinary team, a variety of GMP and clean-room environments, and platforms for lentiviral (LV) and plasmid production complementing the Hub's AAV production.

The NHSBT Gene Therapy Hub will play a key role in the national network of hubs, in which we will collaborate with stakeholders such as LifeArc, Cell and Gene Therapy Catapult (C>C) and the Centre for Process Innovation to deliver research and translation supports to UK academic-led teams.

Within the Hub itself, we will produce AAVs using an innovative platform acquired via technology transfer from C>C, which will be GMP validated at 50L scale. A variety of batch sizes, from 10L to 50L, will be available to suit different research needs. Our GMP environments and processes will be similarly configurable. Production will be complemented by quality assurance, by training (technical, GMP, regulatory, etc.) and by translation-support services, delivered in collaboration with the wider network.

Our NHSBT Gene Therapy Hub will strengthen the UK's position as an international leader in gene therapy research and innovation.