How can we enhance AAV, LV, and non-viral delivery technologies to increase efficiency, tissue targeting, and reduce immunogenicity?
04 Nov 2026
Gene Therapy Development
- 1. Which AAV analytical assays can be performed at low scale and with little to no purification
early in the process of optimizing a new system? Which have you found to be most valuable? - Purification by ultracentrifugation methods have traditionally been used by discovery and
academic groups but these methods may over-estimate the purity that may be achievable by
chromatographic methods. What small scale purification methods have you used during
production optimization that adequately reflect the results you observed during scale-up and
purification? - One way to approach scalable AAV manufacturing is to get experience early on with one of the
non transfection-based methods. What systems have you tried and what was your experience? - What Cell Culture Platforms have been the most predictive of successful scaleup to large
production platforms?