Nikolai Naryshkin
Chief Scientific Officer Ribonaut Therapeutics
Nikolai Naryshkin is Chief Scientific Officer at Ribonaut Therapeutics, with 20+ years targeting RNA with small molecules and nucleic acid-derived therapeutics. At PTC Therapeutics, he led the SMA program that, with the SMA Foundation and Roche, culminated in the approval of Evrysdi (risdiplam) — a first-in-class oral small molecule targeting pre-mRNA splicing — for spinal muscular atrophy. Dr. Naryshkin’s work centers on discovering and developing drug candidates that modulate gene expression in rare genetic and other serious disorders.
Seminars
Thursday 12th November 2026
Roundtable Discussion: Characterizing Functionally Meaningful RNA– Protein Complexes to Enable Productive Drug Discovery
1:30 pm
- What is the minimal RNA–protein complex that recapitulates the functional event we intend to drug – for splicing, is that the initiation/commitment step?
- How should we distinguish binding that is merely observed from engagement of a functionally meaningful fraction of the target RNA population?
- What can structural biology of the functional complex reveal about ligand recognition and the determinants of modulation that structure of the isolated RNA cannot?
Thursday 12th November 2026
Panel Discussion: Establishing RNA-Targeted Small Molecules as a Generalizable Drug Modality
3:30 pm
- 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?
