Leveraging Lessons from Clinical Successes with RNA Splicing Modifiers & Modulators to Improve Small Molecule Drug Design

Time: 3:15 pm
day: Pre-Conference Workshop Day


With Risdiplam being the first RNA-targeting small molecule drug in the clinic, the community is investigating the vast potentials of RNA splicing modulators. As the first-in-class splicing modulator for treatment of spinal muscular atrophy, Risdiplam carved the way to exploring future indications of the chemical space for splicing modulators.

  • How to use RNA splicing to determine new small molecule scaffolds with reduced off-target splicing events
  • Two candidates fell out of clinic due to these off-target events – what can we learn from their downfall?
  • How to design screens to understand how often splicing events occur in nature?
  • Splicing modulators for lead optimization and development at the candidate level