Learn from the companies moving into the clinic, explore emerging modalities beyond splice modulation, and discover how the field is tackling its biggest challenges: selectivity, functional modulation and target validation.
The RNA-targeted small molecule field is reaching a critical inflection point.
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, clinical programs are advancing, pharma interest is growing, and new approaches are expanding what is possible beyond traditional splice modulation.
Join 80+ RNA-targeted drug discovery leaders to:
- Access new data from companies advancing RNA-targeted therapies toward the clinic
- Explore emerging modalities including RNA degraders, covalent binders, and epitranscriptomic approaches
- Tackle key challenges in target selection, functional modulation, and selectivity
- Benchmark strategies with leading biotech, pharma, and academic experts
- Connect with the companies shaping the future of RNA-targeted therapeutic
The 9th RNA-Targeted Drug Discovery & Development Summit brings together leaders from PTC Therapeutics, Remix Therapeutics, ReviR Therapeutics, AstraZeneca, Sanofi, Atomic AI, Syrna Therapeutics, Ribonaut Therapeutics, Ribometrix, and more to share insights, forge partnerships, and accelerate the future of RNA-targeted drug discovery.
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
Why Should I Attend & Why?
Which category do you fit under? See why you should attend this conference:
Large Pharma Leaders
Biotech Decision-Makers
Biotech Drug Developers
Scientists
Academic Researchers
Explore the Full Event Guide
- Connect with 80+ RNA-targeted small molecule leaders from biotech, pharma, and academia
- Hear 18+ case studies spanning discovery, design, validation, and translation
- Participate in 3 interactive workshops tackling the field's biggest challenges
- Discover new insights across splice modulation, direct RNA targeting, degraders, and covalent binders
- Learn how leading teams are approaching screening, selectivity, and target validation
- Join the only conference dedicated exclusively to RNA-targeted small molecules
Meet The Companies Defining The Future Of The Field
The Questions Driving RNA-Targeted Drug Discovery Forward
Why haven't we seen more clinical success stories?
How do we move beyond RNA binding to achieve functional modulation?
Which targets are genuinely therapeutically relevant?
How can we improve selectivity and reduce off-target effects?
What novel chemistries will unlock currently inaccessible RNA targets?
How close are we to broader pharma adoption?
Join the community working to answer these questions.
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Explore the Agenda
Hear the latest industry advances in RNA target selection, structure-guided design, selective modulation and translational strategies across the full RNA-targeted small molecule pipeline and during our packed agenda, interactive roundtables, and panel discussions.
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Join Biopharma Experts
Connect with RNA-targeted small molecule leaders across discovery, preclinical and clinical development during our dedicated networking sessions to exchange insights, build collaborations and strengthen decisions from target selection to therapeutic impact.