What are cell and gene therapies?
Cell and gene therapies are transformative medicines whose full potential is only just beginning to emerge. 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-introduced to 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.
The BIA has a number of members working in the cell and gene therapy space, and we engage with these companies to support the continued growth of the sector and ensure that these treatments can reach patients.
Recent activity in cell and gene therapy
Cell and Gene Therapy Advisory Committee (CGTAC)
The Cell and Gene Therapy Advisory Committee (CGTAC) works 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. It also engages in specific topics to support the development of the cell and gene therapy sector in the UK, including addressing the skills gap, supporting the translation of research and ensuring sustainable patient access to treatments.
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.
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
