Day One

• Assessing RNA targets systematically by defining practical workflows that evaluate functional regions, tractability, binding potential, and risks before screening
• Learning from affinity-based screening pitfalls where compounds bind RNA in biochemical assays but fail to produce meaningful functional activity
• Shifting toward cell-based screens for functional output in RNA-targeted drug discovery

• Predicting RNA structural features that may support selective small-molecule binding
• Distinguishing structurally interesting RNAs from targets with functional therapeutic relevance
• Using model-guided experimental validation to select targets before investing in screening

• Moving beyond case-by-case RNA discovery by developing scalable screening workflows that identify functional RNA structures and tractable targets earlier
• Comparing fragment-based, affinity-based, and cell-based screening approaches to determine which technologies generate actionable RNA-targeted hits
• Prioritizing functional output over binding alone to reduce false positives and identify compounds with meaningful RNA-modulating activity

Put a face to a name – this networking session is your opportunity to get face-to-face with many of

the brightest minds working with RNA-targeted therapeutics to establish meaningful connections to

pursue for the rest of the conference

• Riboswitches provide unique opportunities to systematically explore how RNA productively interacts with a broad chemical space
• Combining in vitro binding, cell-based assays, chemoinformatic modeling and structue-based design, we unmasked a cryptic binding site within the cobalamin riboswitch that was exploited to discover compounds that have affinity exceeding the native ligand
• Our data demonstrate how a privileged biphenyl-like scaffold effectively targets RNA and how other chemistries can be harnessed to achieve high-affinity interactions

Practical and highly interactive breakout roundtables where attendees can crowd

source solutions and share opinions around pre-assigned topic areas.

• Building RNA-focused libraries using known RNA-binding chemotypes, public datasets, chemical similarity, scaffold diversity, and RNA-relevant molecular features
• Addressing limitations of commercial screening collections by reducing reliance on protein-biased, patent-crowded, or overly familiar RNA-binding chemotypes
• Designing ML-interpretable libraries that support more efficient screening, hit clustering, SAR development, and chemical space exploration

Moderator Feedback & Audience Debate

Moderators will be assigned to each roundtable to facilitate discussion and collate

the findings. Following the roundtable discussions, they will present back to the entire

delegation and open wider audience debate

• Building HARIBOSS++, a curated RNA–small molecule binding database that accelerates rational design of RNA-targeted therapeutics
• Detecting binding sites in RNA ensembles with SHAMAN, enabling comprehensive druggability assessment across conformational space
• Parametrizing force fields for RNA dynamics to enhance the accuracy and reliability of computational predictions for drug discovery

• Expanding splice modulation into new RNA sequence contexts to identify novel therapeutic targets
• Decoding how RNA–small molecule interactions drive binding, splice site recognition and modulation mechanisms

• Overcoming challenges while progressing oral RNA splicing modulators into the clinic
• Optimizing activity and selectivity on multiple targets to achieve the best target profile
• Sharing progress on RTX-317 as ReviR advances its next RNA splicing modulator program toward IND-enabling studies

• REM-422, orally available small molecule targeting MYB, is well-tolerated at RP2D (24mg)
• REM-422 shows anti-tumor activity in biomarker positive R/M ACC at RP2D
• REM-422 has indication expansion opportunities in additional solid tumors and hematological malignancies

Immerse yourself in an engaging and informal session, join your peers in a relaxed atmosphere

that encourages meaningful conversations and discussions. Explore a range of exciting poster

presentations and showcase your own developments in the RNA-targeting world. Don’t miss out

on the chance to submit your own posters and get ready to connect, learn, and present. To submit

your poster please contact info@hansonwade.com

• Targeting MYC mRNA through fragment-based small molecule discovery
• Achieving translation knockdown with direct RNA-binding fragments
• Achieving over 70% translation knockdown on various RNAs
• Validating conventional non-covalent approaches for RNA modulation

• Elucidating compound mechanism by defining how mRNA-targeted MYC repression drives modulation of the MYC transcriptional network
• Demonstrating in vivo efficacy to show translation from cellular activity into disease-relevant models
• Enhancing efficacy by refining potency, exposure, and selectivity

• Selectively targeting toxic CUG-repeat RNA in DM1
• Disrupting RNA foci to release MBNL1
• Linking target engagement to restored splicing function
• Building confidence in potency, selectivity and disease relevance