The state-of-the-art Bristol Clinical Biotechnology Centre (CBC) opened at NHS Blood and Transplant’s Filton base in 2023.
The CBC manufactures products for the development of potentially curative therapies for currently incurable diseases, such as some forms of cancer, sickle cell disease and cystic fibrosis.
It supports early phase clinical trials and pre-clinical work, providing a route to eventual commercial scale production.
It will help give patients quick access to the latest treatments by increasing the number of UK patients with incurable diseases who are able to take part in clinical trials and also bring new treatments into the NHS faster.
What are cell and gene therapies?
Cell and gene therapy is a cutting-edge area of medical development. Therapies are based on the idea that living cells or genetic material can be used to cure a wide range of acquired and inherited diseases, by altering their DNA or using them as a vehicle to deliver treatments. Gene and cell therapy can be used to treat illnesses such as leukaemia, haemophilia, autoimmune disorders, cancer, HIV, melanoma, and cystic fibrosis.
Gene therapy works by fixing a genetic problem at its source. Genetic material, usually in a carrier such as a modified and inactivated virus, is transferred to cells, and the faulty DNA is replaced, inactivated, or repaired – for example, gene therapy is being used by the NHS to treat Spinal Muscular Atrophy.
In cell therapy, the patient receives cells which then act with a therapeutic benefit. These cells are often genetically modified– for example, CAR-T therapy, where immune cells are modified to recognise and attack cancer cells. Stem cell transplants or blood transfusions are examples of long-established cell therapies where the cells are unmodified.
Such advances in biotherapies offer new hope for patients for whom all other treatment options have been exhausted. CAR T-cell therapies have now been used to treat hundreds of patients.
How does the CBC help?
The CBC is based at NHS Blood and Transplant (NHSBT) in Filton, North Bristol. Larger commercial sites do exist, which are cost effective for making products for proven treatments. However, researchers need access to flexible sites where they can cost-effectively make smaller amounts for treatments still being researched and clinically tested – that’s what the CBC specialises in.
The UK currently has limited capacity to make the DNA plasmids and viral vectors used in the manufacture of gene therapies and genetically modified cell therapies. In particular, there is insufficient manufacturing at the scale required by organisations wishing to undertake early phase clinical trials of these biotherapies. The shortage of UK manufacturing capacity means long delays for developers of gene therapies while they wait for production slots.
Researchers often need to seek the services of overseas manufacturers, which inevitably delays clinical trials and patients’ access to much needed innovative therapies, and often increases costs. The CBC changes that by expanding the UK’s ability to make its own plasmids and viral vectors.
The new CBC was part funded by a Government grant of £9.43m. Expanding and improving the CBC’s unique offering is in line with the Government’s Invest 2035 Strategy, which identifies advanced manufacturing and life sciences as growth-driving sectors.