Panel Discussion: Establishing RNA-Targeted Small Molecules as a Generalizable Drug Modality
- Which disease areas beyond rare genetic indications – respiratory, immunology, and other high-prevalence settings – are genuinely within reach, and what makes a target there tractable?
- What new chemotypes, mechanisms, and platforms are needed to expand beyond risdiplam- and branaplam-like analogs and a handful of target classes – and how far should we extend the one validated mechanism class versus seek genuinely new ones?
- What translational, safety, and selectivity evidence is required to convince the field that RNA targeting is a generalizable modality rather than a niche?