Panel Discussion: Uncovering Unknowns About the Ratio of Percentage Full Vs Percentage Empty Capsids On Safety & Efficacy of AAV Gene Therapy

Time: 3:15 pm
day: Day One


  • What is the impact of percentage full vs empty ratio on efficacy and safety?
  • What role does this play on immunogenicity of the gene therapy?
  • What are the acceptable limits of empty capsids in a drug product and which assays are use to measure it?

Chaired by: Sam Wandsworth, Chief Scientific Officer, Ultragenyx