Considerations for Translational Dose Selection for Gene Therapies

Time: 11:15 am
day: Day One: Track B AM


  • Reviewing pharmacological concepts for gene therapy and outline a preclinical dose selection research program for gene therapy programs (i.e what needs to be measured in what species NHP vs other species, what are objectives of NHP biodistribution studies)
  • Describing a mathematical model approach to analyzing data from biodistribution studies, with the goal of informing first-in-human gene therapy dose range
  • Illustrating the modeling approach with NHP data