Gene therapy


The Gene Therapy Unit focusses on the most commonly used gene therapy vectors: adenoviral, adeno-associated viral (AAV) and lentiviral vectors. Our goal in vector development programs is to develop safer, regulated and more efficient viral vectors. An understanding of disease at the molecular biology level is key to the way KCT operates and our developed viral vectors are tested in vitro and in vivo models of disease to obtain a clear proof of principle at the molecular biology level.

Translational stage work comprises extensive manufacturing process development, analytical development, mechanistic proof of principle in the target disease and preclinical animal studies. Our early stage gene therapy manufacturing process development and process scale-up encompasses;

- Adenoviral, AAV and lentiviral vectors
- Adherent process platforms (iCELLis technology)
- Suspension process platform (Cultibag and stirred tank bioreactor)
- Clarification, depth filtration, polish filtration
- Chromatographic AEX, affinity purification
- Tangential flow filtration
- Final formulation.