Considerations for Translational Dose Selection for Gene Therapies
Time: 11:15 am
day: Day One: Track B AM
Details:
- 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
