Re-Dosing Discussion Day

Tuesday, August 3 2021 | 10am - 4pm EDT

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

10:00 am PANEL: 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 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

11:30 am 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

12:00 pm PANEL: An Overview & Assessment of Current & Future Strategies to Circumvent or Lessen the Innate & Early Adaptive Immune Response to Gene Therapy

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

1:00 pm Lunch

2:00 pm Round Table Discussion

Synopsis

  • What challenges stand in the way of re-dosing
  • How can these challenges to addressed
  • Strategies to lessen the immune response
  • Strategies to circumvent the immune response
  • Will re-dosing pose a major challenge for gene therapy drug development in the near future?

3:00 pm Round Table Feedback