Gene Therapy

The Gene Therapy team focuses on developing biological and analytical methods to investigate and better understand the safety profile of gene therapy products. Our active projects encompass adeno associated virus (AAV), lentiviral vector (LVV), Chimeric antigen receptor T-cell (CAR-T) therapy and genome edited products. We aim to impact the future quality of gene therapies by addressing regulatory focused research questions and driving the global standardisation and harmonisation of Gene Therapy product critical quality attribute (CQA) assessment through development of novel reference materials.

Additionally, we are 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.

For more information contact claire.kerridge@mhra.gov.uk.

Cell Therapy

The Cell Therapy team conducts regulatory research to advance the safety and reliability of the cell therapy field. We develop biological reference materials that support global standardisation in manufacturing and quality control (QC). Current projects include physical standards for robust phenotypic QC of pluripotent stem cells and mesenchymal stromal cells, as well as reference materials designed to enable accurate and consistent detection of residual PSCs in PSC‑derived cell therapy products.

For further information contact katherine.cornish@mhra.gov.uk.

Nanoparticles

Our team specialises in developing predictive, functional in vitro assays and advanced analytical methodologies for the detection and characterisation of sub‑micron particles, including LNPs, liposomes, and extracellular vesicles/exosomes, as well as for the assessment of Advanced Therapy Medicinal Products, biotherapeutics and diagnostics.

For further information contact anna.nowocin@mhra.gov.uk

UK Stem Cell Bank

The UK Stem Cell Bank (UKSCB) facilitates the use and sharing of quality-controlled human stem cell lines to support scientific research and clinical development of stem cell therapies. We possess state-of-the-art facilities and a growing portfolio of clinical-grade lines and reference reagents available to support the cell-based therapy field.

Find out more here.