Jonathan Schwartz

Company: Rocket Pharmaceuticals

Job title: Chief Medical Officer

Seminars:

Optimizing AAV Clinical Safety: Evolving Immunomodulation in the Phase 1 Study of RP-A501 (AAV9.LAMP2B) in Danon Disease 2:30 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 cohortRead more

day: Day 1 Tracks 2 PM

Industry Leaders Panel Discussion: Addressing High Dose AAV Delivery 9:30 am

Have we reached the highest dose for gene therapy yet? Exploring dosing strategies: “One and done or Slower and Lower?” What impact would a cumulative vector genome delivery approach have on safety and efficacy? Moving away from one and done: what does best practice look like for dosing strategies? Which role does complement play in…Read more

day: Day One