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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
  • 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?
Industry Expert
William Perry, Distinguished Scientist Director - Johnson & Johnson