AAV Programme – European Biotech

eXmoor delivered a complete AAV process for a pre-clinical gene therapy company focussed on neurodegenerative diseases.

The initial research process needed to be scaled-up and optimised to meet target dose requirements and GMP standards. eXmoor carried out a full process development package including cell line, media and clarification filter screening as well as transfection, culture conditions, and anion exchange chromatography optimisation using DoE modelling.

Both viral genome yield and final Full:Empty ratio were increased 5-fold in the final process which was scaled-up to a low-endotoxin 50 L batch destined to dose finding studies. The client is currently undergoing clinical trials.