9am - 4.10pm EDT

PRE-EXISTING IMMUNITY & CLINICAL TRIAL ELIGIBILITY

9:00 am Pre-Existing Anti-AAV Antibodies: What’s The Problem? What’s The Solution?

Synopsis

  • How to define the seroprevalence and titer of pre-existing antibodies that vary with age and geographical location
  • What solutions are currently available to reduce the titers of pre-existing antibodies?
  • The logistical challenges of reducing the titers of pre-existing antibodies

9:30 am Immunogenicity Prediction and Dose Optimization using Proven In Silico Modeling & Simulation

Synopsis

• FDA’s risk based guidance for managing IG is focusing on the use of modeling & simulation for proteins, vaccines and new modalities
• This immunogenicity modeling technology is being applied to address the unmet needs emerging from advanced therapies, specifically gene therapy
• A case study demonstrating the use of this approach for dose optimization for gene therapy will be shared

9:45 am Immunogenicity Risk Assessment For AAV-Based Gene Therapies

Synopsis

  • Which criteria should be taken into account for the immunogenicity risk assessment of AAV-based gene therapies?
  • Which assay systems are available or in development to evaluate those risks?

10:15 am Tools for Immunogenicity Assessment of Gene Therapy Products

Synopsis

• Assessing unwanted immunogenicity early on using in silico and in vitro tools can de-risk Gene Therapy products
• In vitro Elispot/Fluorospot assays as a powerful tool for monitoring cellular immune responses of Gene Therapy
• The importance of PBMC quality during cellular immune assays

10:45 am Have The Speakers Answer Your Questions

11:15 am Morning Break & Speed Networking

11:45 am PANEL: Data Sharing in the Context of Conquering Immune Response to Gene Therapy Delivery

  • Susan Ward Founder & Executive Director, cTAP - Duchenne
  • Guangping Gao Professor & Director, UMass Medical School

IMMUNOGENICITY IN THE CLINIC

12:30 pm The Impact Of Innate & Anti-Transgene Immunogenicity On AAV Vector- Mediated Gene Transfer

Synopsis

  • Introducing clinical experience on the immunogenicity of AAV capsids
  • Innate and anti-transgene immune responses to the vector product
  • Recent advances and future perspectives for a safe and efficient gene transfer in humans

1:00 pm Development of an Immunogenicity Risk Assessment Strategy to Drive Bioanalysis & Clinical Immune Monitoring for Viral Gene therapies

  • Vibha Jawa Executive Director , Bristol Myers Squibb

Synopsis

  • Risk assessment of gene therapy based biologics: understanding risks due to capsid serotypes, transgene, site of administration, standard of care treatments and dose, product and host related factors
  • Bioanalytical assays for PK and immunogenicity and clinical and nonclinical studies
  • Assay formats and technical challenges

1:30 pm Visit Partner Demo Area

2:30 pm Lessons From Cardiac AAV-Mediated Gene Therapy

Synopsis

  • Perspective of targeting the myocardium
  • Addressing the immune response to AAV
  • Next steps in advancing the supportive care of AAV gene therapy

3:00 pm Have The Speakers Answer Your Questions

OPTIMIZATION OF CMC PROCESSES TO REDUCE IMMUNOGENIC RESPONSE

3:30 pm Interpreting Regulatory Guidances to Help Drive CMC Optimization Activities

  • Maria Lobikin Associate Director, CMC Regulatory Affairs, Homology Medicines

4:10 pm DAY END