Protocol Design of Early Stage AAV Clinical Trials for Safety Optimization

Time: 9:30 am
day: Day Two


  • Uncover how AAV vectors (even within the same serotype) demonstrate significant diversity in the immune responses elicited and their clinical risk
  • Explore how clinically significant adverse events have been observed with AAV vectors involving innate and adaptive immunity, specifically complement, leukocyte and cytokine activation, T cell responses, and humoral immunity
  • Uncover all available information, including previously communicated trials and experience, vector properties, and in vitro and in vivo preclinical studies should inform immunosuppression strategies