Lessons Learned from AstraZeneca’s Drug Discovery Projects: Workflow, Pitfalls, & Screening Technologies to Prioritize Functional RNA-Targeted Hits

  • Assessing RNA targets systematically by defining practical workflows that evaluate functional regions, tractability, binding potential, and risks before screening
  • Learning from affinity-based screening pitfalls where compounds bind RNA in biochemical assays but fail to produce meaningful functional activity
  • Shifting toward cell-based screens for functional output in RNA-targeted drug discovery