Small Molecules Targeting RNA Expression: Evaluation of Preclinical Therapeutics in Pathophysiological Models of Disease

Time: 2:20 pm
day: Day 1 Track B PM


  • Examining translational medicine approaches to demonstrate human efficacy in vitro by utilizing IPSC models that asess small molecule RNA therapeutics for Huntington’s disease
  • Leveraging unique ‘omics analyses to demonstrate robust target engagement and target specificity profile to enhance translational confidence to the clinic
  • Limitations and challenges when utilizing patient derived models in vitro vs. in vivo assessments followed by direct omic comparisons