Approach for Modeling and Species-Translation of Biodistribution, Transduction and Efficacy of AAV-based Gene Therapies

Time: 10:00 am
day: Day Two


  • Data-driven identification of Dose, Serotype, Construct, Target tissue and Species dependencies of AAV treatment response
  • Role of modeling in preclinical evaluation of AAV gene therapies – optimizing biodistribution study designs
  • Use QSP modeling frameworks for species translation and minimum effective dose determination