Harnessing RNA Interaction Insights, Improving Functional Modulation & Uncovering Translational Strategies to Unlock Selective, Effective RNA-Targeting Small Molecules for Improved Treatments of Oncological, Rare Genetic & Neurodegenerative Indications
RNA-targeting small molecules are rapidly gaining therapeutic traction, underscored by clinical advances including ReviR Therapeutics first participant dosed in a Phase 1 study of RTX-117 and Remix Therapeutics reporting positive Phase 1/2 data for REM-422. Building on the clinical validation of splice modulation, the field is progressing across direct RNA binding, RNA degradation, covalent translation inhibition, epitranscriptomic regulation and condensate biology, establishing RNA-targeted small molecules as an increasingly credible route to modulate disease biology once considered beyond the reach of conventional protein-targeted strategies.
With scientific innovation at the core of this momentum, the 9th RNA-Targeted Drug Discovery & Development Summit returns to Boston this November as the industry’s only dedicated forum focused exclusively on RNA-targeted small molecules. Bringing together leaders from pioneering biotechs, major pharma and expert academia, including PTC Therapeutics, Remix Therapeutics, ReviR Therapeutics, AstraZeneca, Sanofi, Atomic AI, Syrna Therapeutics, Dewpoint Therapeutics, EPICS Therapeutics, Ribonaut Therapeutics and Ribometrix, the summit will deliver the latest data, practical discovery strategies and collaborative discussion shaping the future of RNA-targeted therapeutics.
With a refreshed agenda spanning functional RNA target selection, structure-guided design, productive chemical space, emerging RNA-targeting technologies, and translational considerations, this forum reaffirms its position as the go-to meeting for connecting the scientific and strategic decisions needed to progress RNA-targeted small molecules from discovery toward clinical impact.
The meeting was exceptionally well organized, and I truly enjoyed the depth and openness of the scientific discussions.
What stood out to me was the strong balance between fundamental RNA biology, emerging technologies, and translational perspectives across pharma and biotech.
The highly engaged audience and the frank exchange of technical challenges made the sessions particularly valuable.
It was also inspiring to see how different groups are approaching RNA-targeted small molecules from diverse angles, which made the conversations both enjoyable and thought-provoking.
Co-Founder, Representative Director, President & Chief Executive Officer, xFOREST Therapeutics
I greatly enjoyed the panel discussions, which were highly interactive and offered different perspectives from leaders in the field. It was particularly exciting to learn about the new chemical entities currently in clinical trials, as well as the novel strategies for RNA modulation and tools for discovery. Overall, the summit was both informative and inspiring, giving me valuable insights and ideas that I can bring back to my own work.
Chief Scientific Officer, Veritas In Silico
RNA-Targeted Drug Discovery Summit has become the main annual event for RNA centric small molecule drug discovery community. It is the most representative congress to learn about the progress in the field, connect with colleagues from other companies and laboratories.
Chief Scientific Officer, Ribonaut Therapeutics
This meeting is the main chance each year for all of the thought leaders in drugging RNA with small molecules to get in one room and exchange ideas. If you want to know what the latest approaches are to cracking the RNA-drugging problem, you’ll find them here.
Chief Technology Officer, Wayfinder Biosciences
We are entering an exciting era in which RNA is becoming a tractable and increasingly diverse therapeutic target. Realizing its full potential requires contributions from both academia and industry - from understanding RNA structure, function, and druggability to advancing compounds toward the clinic. Meetings like this one are invaluable for fostering the exchange of ideas and collaborations needed to drive the next generation of RNA-targeted medicines
Director & Professor - Medicinal Chemistry, University of Toronto
Explore the Full Event Guide
- Connect With 80+ RNA-Targeted Small Molecule Experts Including PTC Therapeutics, Remix Therapeutics, Arrakis Therapeutics, AstraZeneca, Sanofi and more
- Hear From 18+ Expert-Led Case Studies Spanning RNA Target Discovery, Chemistry, Optimization, Validation and Translation
- Join 3 Interactive Workshops on Functional RNA Target Selection, RNA Structure Probing and RNA-Focused Chemical Space
- Access New Data Across Splice Modulation, Direct RNA Binding, Fragment Discovery, Covalent Translation Inhibition and Kinetic Characterization
- Attend Dedicated Discussions Tackling How to Select the Right Screening Strategy, Build RNA-Focused Libraries, Measure Selectivity, and more
- Be Part Of The Industry’s Only Dedicated Forum for RNA-Targeted Small Molecules
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