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
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.
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.
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
Dr Sven Kili is currently principle at Sven Kili Consulting Ltd. Where he provides specialist strategic advice to Regenerative Medicine companies. He was previously the VP and Head of Development for the Cell and Gene Therapy division of GSK Rare Diseases where he led teams developing ex-vivo Gene Therapies for a variety of rare genetic disorders including Strimvelis®, the first ex-vivo gene therapy to be approved for children with ADA-SCID; Wiskott - Aldrich syndrome (WAS); Metachromatic Leukodystrophy (MLD) and Beta-Thalassemia.
Prior to this, he was Senior Director, Cell Therapy and Regenerative Medicine for Sanofi (Genzyme) Biosurgery where he led the clinical development and medical affairs activities culminating in the granting of the first combined ATMP approval in the EU for MACI®. His team also prepared and submitted Advanced Therapy regulatory filings for Australia and the US, including health technology assessments and he was responsible for late stage developments for Carticel® and Epicel® in the US.
Before joining Genzyme, Sven worked for Geistlich Pharma where, in addition to leading the cell therapy activities, he oversaw all UK & Irish regulatory functions and was the QPPV for pharmacovigilance for the EU.
Sven trained as an Orthopaedic surgeon in the UK and South Africa and since leaving full-time clinical practise has developed expertise Cell and Gene Therapy in clinical development, regulatory compliance, value creation, risk management and product safety,
Helen Delahaye
Helen Delahaye is currently Managing Director at Deltohn Ltd, a consultancy which provides interim management to a number of biopharmaceutical clients with the aim to assist companies to improve efficiency, productivity and profitability. She has extensive strategic and technical knowledge of the drug development process spanning over 20 years and covering research, development and commercialisation. Prior to this, Helen has held senior management positions at Quintiles, Huntingdon Life Sciences and ProStrakan.
CGTAC members
