Optimizing AAV Clinical Safety: Evolving Immunomodulation in the Phase 1 Study of RP-A501 (AAV9.LAMP2B) in Danon Disease

Time: 2:00 pm
day: Day 1 Tracks 2 PM


  • Overview of immunologic side effects associated with systemic AAV therapy
  • Review of the safety and preliminary efficacy in the adult and pediatric cohorts of the Phase 1 Danon disease study
  • Overview of complement activation and enhancements in the immunomodulatory regimen for the pediatric cohort