NHS Blood and Transplant (NHSBT) has opened a state-of-the-art facility which will contribute greatly to the UK’s ability to develop and manufacture new gene and cell therapies.
It will manufacture products for the development of potentially curative therapies for currently incurable diseases, such as some forms of cancer, sickle cell disease, and cystic fibrosis. Some of these will be personalised therapies, aimed at treating just one person. The new Clinical Biotechnology Centre (CBC), which has been built in Bristol with a near £10m Government grant, is designed to expand the UK’s ability to make the clinical grade products required for the research and development of new cell and gene therapies. It will support 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.
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 example 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.
The CBC has been built at NHSBT’s base in Filton, in North Bristol, and replaces a smaller, ageing unit in nearby Langford. 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 will specialise 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 new CBC will change that by expanding the UK’s ability to make its own plasmids and viral vectors.
The CBC expansion 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 Life Sciences Industrial Strategy, which is to grow the UK’s manufacturing capacity for DNA-based therapeutics.
Steve Barclay, Health and Social Care Secretary, said: “The government is backing new gene and cell therapy which uses living cells or genetic material such as DNA to cure diseases. The investment in this new centre will continue to develop this area of research and help provide patients the best possible care.
“We announced our ambitious Life Sciences Industrial Strategy to tackle cancer and life-changing illnesses and ultimately save lives. This works best when we work collaboratively and this new centre is the perfect example of the government, NHS and those in research and manufacturing collaborating to eventually deliver new transformative treatment to NHS patients.”
Steve Bates, Chief Executive Officer of the UK BioIndustry Association, said: “NHS Blood and Transplant is something of a hidden secret in the UK ecosystem in terms of its capability to manufacture cell and gene therapies. This fantastic new centre will enable their capable team to better partner with companies in this key growth area of our life science economy.”