Manufacturing of AAV vectors: translational challenges from development to industrialisation

AAV mediated gene therapy offers the potential for curative therapies for many fatal and debilitating diseases, however the translation of these concepts into licenced products is stymied by manufacturing challenges leading to very high costs and delayed commercialisation. This is an issue which vector developers need to address, specifically around productivities and vector purities including levels of empty non-functional capsids. To date vector developers have focused on the optimising of vector targeting and gene delivery, however there is an increasing need to recognise the issue of manufacturability of vectors, and to be able develop screening and assessment of novel vectors to ensure that in future innovations and development can be produced to the required quality and in the desired quantities and at an acceptable cost.