Explore the Agenda
7:30 am Check-In & Morning Coffee
8:15 am Chair’s Opening Remarks
8:29 am Improving RNA-Targeted Lead Design Through Structure-Guided Covalent Chemistry & Mechanistic Deconvolution
8:30 am Rational, Structure-Guided Design of Small Molecule Covalent Translation Inhibitors Targeting mRNA
- Identifying structured regions in mRNA as small molecule targets using cryoEM
- Using 3D information to locate small molecule binding site and understand effect of small molecule binding on RNA structure
- Dependence of translation inhibition on binding site induced proximity and proper orientation of electrophile within binding site
- Importance of using cooler electrophiles when designing covalent translation inhibitors
9:00 am Panel Discussion: Target-Focused vs High-Throughput Approaches for Discovering Therapeutically Effective RNA-Targeting Small Molecules
- What are the advantages and challenges associated with target-focused approaches?
- What are the advantages and challenges associated with high-throughput phenotypic or cell-based functional screening approaches?
- How is phenotypic RNA modulation connected back to a specific RNA target, binding site, or mechanism of action?
- How does each approach manage generating actionable SAR and program attrition?
9:45 am Morning Break & Networking
10:44 am De-Risking RNA-Targeted Small Molecule Progression by Assessing Selectivity, Safety & In Vivo Validation
10:45 am Positioning Long Non-Coding RNAs as Druggable Cancer Targets Through Small Molecule Modulation
- Exploring lncRNAs as actionable targets in prostate cancer
- Targeting SChLAP1 with selective small molecule approaches
- Sharing emerging toxicity and xenograft data in vivo
11:15 am Roundtable Discussion: Measuring Selectivity Across RNA-Targeted Small Molecule Approaches to Build Functional Confidence
- Defining selectivity across different RNA-targeting mechanisms
- Understanding sequence, structure, and mechanism-driven selectivity
- Deciding what “selective enough” means for progression
12:00 pm Lunch & Networking
12:59 pm Linking RNA Target Engagement, Kinetics & Functional Complex Biology to Drug Discovery Decisions
1:00 pm Advancing RNA-Targeted Drug Discovery Through Biophysical & Kinetic Characterization of Small Molecule/RNA Interactions
- Applying single-molecule and kinetic methods to characterize RNA–small moleculeminteractions in depth
- Defining the precise molecular species bound by RNA-targeting drugs to strengthen assay design and screening strategies
- Translating mechanistic insights into more informed hit identification, target engagement studies, and therapeutic development decisions
1:30 pm Roundtable Discussion: Characterizing Functionally Meaningful RNA– Protein Complexes to Enable Productive Drug Discovery
- 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?
2:00 pm Afternoon Break & Networking
2:29 pm The Next Frontier for RNA-Targeted Drug Discovery: Emerging Approaches to Unlock New Biology & Expand Druggable Target Space
2:30 pm Advancing First-in-Class Condensate Modulators for Previously Inaccessible Targets
- Introducing Dewpoint’s condensate biology platform and its application to the discovery of first-in-class therapeutics across oncology and other disease areas
- Highlighting progress across lead condensate-modulating programs targeting historically inaccessible disease drivers
- Exploring emerging insights into RNA and condensate biology to inform future therapeutic opportunities
3:00 pm RNA Methyltransferase Inhibition Validates Epitranscriptomic Regulation as a Novel Approach in the Treatment of Solid Tumors
- Positioning RNA epitranscriptomics as a novel small molecule modality for cancer therapy
- Exploring methyltransferase inhibition as a means to alter tumour cell phenotype and limit cancer adaptability
- Mechanisms underlying the pharmacology of METTL3 inhibition across tumor types
3:30 pm 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?