IQ Webinar: Immunogenicity of Adeno-Associated Virus (AAV) Based Gene Therapies
March 25, 2021
Webinar
Immunogenicity of Adeno-Associated Virus (AAV) Based Gene Therapies: Risk Assessment and Bioanalytical Considerations
Presenters: Tong-yuan Yang, PhD (Janssen R&D), Steve DeWall, Ph.D. (Spark Tx), and John Kamerud, PhD (Pfizer)
Sponsored by the IQ Translational and ADME Sciences Cell and Gene/Nucleic Acid Based Modalities Working Group
The immunogenicity assessment of AAV-based vector gene therapies (GT) is a crucial aspect of drug development that would be critical to understand both safety and efficacy aspects of the modality and is a component of the nonclinical and clinical submission package required by regulatory agencies prior to approval.
This public webinar will provide an overview of the in-depth discussions that occurred as part of the IQ TALG Working Group on Novel Modalities. The following topics will be covered:
- Immunogenicity risk assessment. Some considerations include:
- Product- and host-related risk factors
- Engagement of the innate and adaptive immune pathways; suggests complex nature of immunogenicity associated with GTs
- Regulatory expectations on assessing immunogenicity during nonclinical and clinical studies
- Current learnings on immunogenicity of GTs related to efficacy, persistence, and immunotoxicity
- Clinical mitigation strategies for immunogenicity
- Bioanalytical considerations/strategies during nonclinical and clinical development for monitoring immunogenicity
- Bioanalytical methodologies applied to assess immunogenicity in nonclinical and clinical studies:
- Proposed assay format and technical challenges during assay development
- Considerations in critical reagent needs
- Sample collection, handling, and data interpretation/reporting
- Assay validation strategies
This webinar will provide a comprehensive overview of immunogenicity risk assessment and bioanalytical considerations and current learning of immunogenicity for AAV based GTs. Speakers will share portions of IQ working group discussions especially where additional clarity is needed around the development and validation of novel bioanalytical methodologies and data interpretation.