Cell and gene therapy

Cell and gene therapies are a transformative new category of medicines whose full potential is only just beginning to emerge. Standard, small-molecule treatments are produced in a standardised fashion, and most are relatively short-lived within the body. 

Cell and gene therapies are different. They involve extracting cells, protein or genetic material (DNA) from the patient (or a donor), and altering them to provide a highly personalised therapy, which is re-injected into the patient. Cell and gene therapies may offer longer-lasting effects than traditional medicines. They have the potential to address complex diseases, such as motor neurone disease, and many rare disorders for which there are no effective treatments.

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UK cell and gene therapy report 

The UK has played a major role in the research and development of cell and gene therapies, staking a claim as a leader in these treatments. This leadership is demonstrated by the number of companies based in the UK, the significant financing the UK-based companies have attracted over the past five years, and the high volume of clinical trials taking place in the UK. While the figures for both financing and clinical trials have slowed in recent years – mirroring trends seen across the life sciences sector – the UK continues to be the European leader for cell and gene therapy development.

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Cell and gene therapies explained

To learn more about Cell and Gene Therapies, download our Cell and Gene Therapies Explained: A guide to cell and gene therapy and UK excellence in the field, featuring case studies from BIA members Cell Medica, Immunocore, Nightstar Therapeutics, Horizon Discovery and Oxford Biomedica. You can download the report below.

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Ensuring patient access to cell and gene therapies

Cell and gene therapies are already demonstrating their value and living up to the promise of delivering transformative outcomes for patients living with leukaemia, Spinal Muscular Atrophy and other diseases. Over the next few years, the number of cell and gene therapies is set to grow and more patients will soon be able to benefit from this innovation.

Cell and gene therapies face particular challenges within the evaluation and reimbursement system owing to their high up-front cost and uncertainty with regard to long-term outcomes. Innovative payment models are already being explored to secure access to cell and gene therapies and to balance affordability, sustainability and risk between NHS and industry. 

Leading Innovation|The UK's ATMP Landscape

The Alliance for Regenerative Medicine (ARM) and the BioIndustry Association (BIA) have published a report setting out the scope of the UK’s robust and growing Advanced Therapy Medicinal Products (ATMPs) sector which includes the development of potentially transformative cell and gene therapies.

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Celebrating UK bioscience | Cell and gene therapy

We have also produced a video alongside the explainer which features case studies from Oxford Biomedica and the Cell and Gene Therapy Catapult.  This video explores BIA member companies working on researching, developing and manufacturing the very latest cell and gene therapies. 

We would like to thank the BIA's Cell and Gene Therapy Advisory Committee for supporting the production of the video and guide.