Next Generation Splicing Modulators

Time: 11:00 am
day: Day Two


  • The approval of Risdiplam for the treatment of SMA validated the approach of selectively modulating splicing with small molecules
  • PTC has developed a comprehensive discovery platform to identify novel splicing modulators for the treatment of diseases with unmet needs
  • Novel splicing modulators are being optimized for recently identified disease-relevant splicing events