Gene therapy is one of the most complex human medicines because most gene medicines have combined properties of viruses, bacteria, nucleic acids, recombinant proteins and, in some cases, cells.
This complexity makes product control and standardisation especially difficult.
Also, manufacturers’ testing methods are often product-specific and are not standardised using harmonised reference materials or standards. This makes it difficult to make reliable comparisons of production batches and to evaluate materials used in clinical trials.
The three areas of activity in our section – standardisation, control and research and development (R&D) – are closely interrelated, with our current focus on R&D.
This is because at present there is only one marketing-authorised gene therapy product in Europe.
Our current activities include establishing reference materials to safeguard clinical products such as reference materials for detecting unwanted replication-competent viruses.
We are also developing suitable biological and analytical methods to monitor the safety of advanced gene therapy products – including products based on plasmid DNA, adeno-associated virus (AAV), lentiviral vectors and genetically-modified cells.
We run an active R&D work programme aimed at understanding what factors govern product quality and performance in a production process. This enables us to establish meaningful control parameters, criteria and procedures for the characterisation of gene therapy products. It also means we can establish suitable models to examine and to predict product safety.
NIBSC is a member of the European Official Medicine Control Laboratories (OMCL) gene therapy network which aims to establish and harmonise reference materials and testing methods across the EU for gene therapy products.
We also contribute to the European Medicines Agency Committee for Advanced Therapies (EMA/CAT) work programme. This includes developing European guidance and policies for gene therapy products.
NIBSC contributes to the World Health Organisation (WHO), ISO and International Conference on Harmonisation guidance for gene therapy.
Stifani Satkunanathan - Senior Scientist