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
