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.

What is 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.

UK cell and gene therapy report

Leading the path to transformative medicine

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.

 
Cell and gene therapies explained

Cell and gene therapies explained

As engineers and biologists join forces to build evermore sophisticated gene-editing tools, new classes of medicines are emerging, including cell and gene therapies. These involve altering cells or genes, usually outside the body, to provide a patient-specific therapy that is re-injected into the patient. Learn more about the UK excellence in the field and read case studies from BIA members Cell Medica, Immunocore, Nightstar Therapeutics, Horizon Discovery and Oxford Biomedica.

Read the report

 

Ensuring patient access to cell and gene therapies

Ensuring patient access to cell and gene therapies

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

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Cell and Gene Therapy Advisory Committee (CGTAC)

The Cell and Gene Therapy Advisory Committee (CGTAC) was established to highlight the value of cell and gene therapies to patients and the wider UK economy and to promote the UK’s global role in delivering these transformative treatments and the innovative science behind them.

The committee brings together a large proportion of the UK’s regenerative medicine industry, including biotechs, pharma, service providers, law firms and venture funds, as well as key organisations including the Cell and Gene Therapy Catapult and Innovate UK as members.

CGTAC priorities
1. Connect
  • Promote UK excellence in cell and gene therapies (CGT) both internationally and with UK policymakers
  • Build on strong partnerships with ARM, FIRM and commercial meeting organisers such as Phacilitate, and seek opportunities to engage with other coalitions
  • Build connections with the regulatory environment, including the MHRA, EMA and FDA
2. Inform/Support:
  • Highlight the UK’s leading position in CGT by updating the report series published with ARM
  • Provide guidance on the UK’s CGT infrastructure by developing a roadmap of groups and initiatives
  • Present information on supply chain issues for CGT and how these might be resolved
  • Keep CGT central to government policy, by supporting BIA engagement with government and Parliament
3. Expert advice
  • Respond to opportunities to engage with the NHS on access and reimbursement policies for CGT, including the Innovative Medicines Fund and patient registries
  • Play a leading role in the continued development of skills and training for CGT, including promotion of relevant qualifications and courses
  • Input to the development of measurements and standards for CGT via the MHRA, the National Measurement System, the British Standards Institute and the Standards Coordinating Body
CGTAC Chair and Vice-Chair
Sven Kili

Sven Kili

Principle, Sven Kili Consulting Ltd

Helen Delahaye

Helen Delahaye

Managing Director, Deltohn Ltd

CGTAC members