Establishing Core Endpoints That Truly Correlate with Biological Readouts to Validate mRNA Functionality

  • Comparing in vitro, cell-based and engineered model systems to determine which assays should be standardized to best represent functional mRNA performance
  • Differentiating between integrity, encapsulation, and total mRNA content to determine which attributes meaningfully correlate with biological activity
  • Lessons learnt from CQAs that failed to predict immunogenicity or efficacy to streamline submission packages that prove mRNA functionality