Whitepaper
Whether you’re designing early-phase protocols or preparing for global filings, this guide provides actionable insights to strengthen your assay strategy and advance your gene therapy program with confidence.
Understanding Immunogenicity and AAV Gene Therapy
Best Practices for Designing Robust Assays and Navigating Regulatory Pathways
As AAV-based gene therapies advance into clinical development, managing immunogenicity risk has become essential to ensuring patient eligibility, therapeutic durability, and regulatory approval. This expert-authored white paper delivers a practical, science-backed framework for building immunogenicity assays that are analytically rigorous, clinically relevant, and globally aligned.
What You’ll Learn:
- TAb vs. NAb Assays: In-depth comparison of formats, throughput, sensitivity, and translational relevance
- Assay Strategy Across Phases: Guidance on assay selection and validation from preclinical studies to CTA and companion diagnostic development
- Navigating Global Regulations: Insights into evolving regulatory expectations in the US (IDE, SRD, Q-submissions) and EU (IVDR)
- Validation Best Practices: Technical considerations for cutoff determination, reagent qualification, and global deployment
- Linking Immunogenicity & Efficacy: Approaches for integrating immune response data with therapeutic endpoints to refine patient stratification and study design
Whether you’re designing early-phase protocols or preparing for global filings, this guide provides actionable insights to strengthen your assay strategy and advance your gene therapy program with confidence.
.png?width=558&height=722&name=Screenshot%20(895).png)