How to Design & Validate Potency Assays for Hard-to-Transduce Serotypes & Tissue-Specific Promoters Against Real-World Constraints

  • Designing functional potency assays that reflect true mode of action, using enzyme-based or activity-driven endpoints embedded within cell-based systems
  • Implementing layered control strategies to manage variability across complex cell-based potency assays to enable qualification, validation, and long-term assay stability
  • Reviewing strategies for managing assay life cycle