A new Clinical Biotechnology Centre (CBC) has opened in Bristol to expand the UK’s ability to develop and manufacture new gene and cell therapies.
The Centre will make products for the development of potentially curative therapies for currently incurable diseases, such as some forms of cancer, sickle cell disease, & cystic fibrosis. Some will be personalised therapies, aimed at treating just one person.
Run by NHS Blood and Transplant and backed with a £10m Government grant, the Centre will support early phase clinical trials & pre-clinical work, providing a route to eventual commercial scale production.
It will provide 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 therapies are based on the idea that living cells or genetic material can be used to cure a wide range of acquired & inherited diseases, by altering their DNA or using them as a vehicle to deliver treatments. Gene & cell therapy can be used to treat illnesses such as leukaemia, haemophilia, autoimmune disorders, cancer, HIV, melanoma, & 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.1 Another example of Libmeldy, used to treat metachromatic leukodystrophy.2
- 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.3 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 replaces a smaller, ageing unit nearby. 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.4
The expansion is in line with the Government’s Life Sciences Industrial Strategy to grow the UK’s manufacturing capacity for DNA-based therapeutics.
‘Free of disease now’
Nitya Raghava, 22, from Gloucester, had lifesaving CAR-T cell therapy for acute lymphoblastic leukaemia.
She had relapsed after treatment, and CAR-T was “pretty much a last resort.”
Nitya went on to become the first person to receive CAR-T therapy at the Bristol Haematology and Oncology Centre in February 2019, and has been free of the disease for four years.
She said: “CAR-T was absolutely lifesaving for me. Without it, I don’t think I would be here.
“I think it’s just so exciting to see other new cell and gene therapies being developed at the CBC that can help other people too.
“I now feel great, I’m at university and I am living my life as normal. four years on from receiving CAR-T cells, because I’m in complete molecular remission with no evidence of disease now.
Dr Lilian Hook, NHSBT’s Director of Cell, Apheresis and Gene Therapies said: “The CBC is basically a factory – it manufactures the building blocks (or components) needed to produce gene therapies. Researchers and developers can ask us to manufacture the specific components they require.
“The CBC will help the UK grow its cell and gene therapy industry in a rapidly growing international market. We won’t be designing the treatments, but we will be manufacturing them to the right scale and clinical grade. Cell and gene therapy is a growing area for the healthcare sector and part of our direction of travel as an organisation.”
Steve Bates, of the UK BioIndustry Association, added: “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.”
ReferenceS:
Toru Takumi, VR-based real-time imaging reveals abnormal cortical dynamics during behavioral transitions in a mouse model of autism, Cell Reports (2023). DOI: 10.1016/j.celrep.2023.112258. www.cell.com/cell-reports/full … 2211-1247(23)00269-3
https://www.england.nhs.uk/2021/03/nhs-england-strikes-deal-on-life-saving-gene-therapy-drug-that-can-help-babies-with-rare-genetic-disease-move-and-walk/
https://www.england.nhs.uk/2023/02/first-baby-receives-life-saving-gene-therapy-on-nhs/
https://www.england.nhs.uk/cancer/cdf/car-t-therapy/
The CBC will make:
Plasmids – small fragments of DNA engineered to introduce gene(s) of interest into cells. Their plasmids may be used for direct vaccination of patients (e.g. for HIV infection), cell therapies, or as the starting material for viral vectors.
Viral Vectors – viruses modified to deliver DNA into cells, and commonly used in gene therapy for cell re-programming for a therapeutic effect.
Recombinant proteins – proteins with DNA combined from two or more sources. Made by cells whose DNA has been altered, they have a wide range of uses including as medicines.
