Re-Dosing Discussion Day

Tuesday, August 3 2021 | 10am - 1.45pm EDT

SESSION 1: WHY DO WE CARE ABOUT RE-DOSING?

10:00 am An Introduction to Gene Therapy Delivery & Immune Response & It’s Implications for Re-Dosing

SESSION 2: WHAT ARE THE IMMUNOLOGICAL & THERAPEUTIC IMPLICATIONS OF RE-DOSING?

10:30 am An Overview Of AAV Capsid Neutralizing Antibodies & Associated Complement Mediated Immunotoxicties

Synopsis

  • Reviewing preclinical and clinical data on the links between innate and humoral capsid immunity
  • Pre-existing and acquired capsid NAbs – cross-reactivity, making sense of in vitro NAb assays, strategies to overcome NAbs
  • Complement mediate immunotoxicities with higher vector doses

SESSION 3: STRATEGIES TO CIRCUMVENT OR LESSEN THE INNATE & EARLY ADAPTIVE IMMUNE RESPONSE FOLLOWING GENE THERAPY DELIVERY

11:00 am Strategies to Remove the Barrier of Humoral Immunity in AAV Dosing and Re-dosing

11:30 am Mitigation Of AAV Vector Immunogenicity With Tolerogenic Immtor Nanoparticles

Synopsis

  • ImmTOR nanoparticles encapsulating rapamycin induce antigen-specific immune tolerance
  • Enhancement of liver-directed transgene expression observed at both initial and repeat doses of AAV vectors
  • ImmTOR nanoparticles currently in Phase 3 trials with an immunogenic uricase enzyme therapy for the treatment of refractory gout

12:00 pm Immune Evasive AAV for Repeat Dosing

Synopsis

  • Current AAV technology advantages and disadvantages
  • Data showing Chameleon’s EVADER technology repeat dosing in mice
  • Next steps: EVADER development

SESSION 4: WHAT HAVE WE LEARNT & HOW DO WE TAKE THIS FORWARD?

12:30 pm PANEL Q&A: An Overview & Assessment of Current & Future Strategies to Circumvent or Lessen the Innate & Early Adaptive Immune Response to Gene Therapy