Why RNA-Targeted Drug Discovery Matters More Than Ever
The RNA-targeted small molecule field is entering a pivotal stage of growth.
RNA-targeted small molecules are rapidly evolving from a promising scientific concept into a clinically relevant therapeutic modality. Following the success of splice modulation and the approval of risdiplam, a growing number of companies are advancing RNA-targeted programs into the clinic, generating new data, securing major partnerships, and expanding the therapeutic potential of RNA-directed medicines.
Yet significant challenges remain. The 9th RNA-Targeted Drug Discovery & Development Summit brings together the biotech pioneers, pharma leaders, and academic innovators driving the field forward. As the industry's only conference dedicated exclusively to RNA-targeted small molecules, it provides a unique forum to share new data, benchmark strategies, discuss challenges openly, and build the partnerships needed to accelerate progress.
Join leading organizations including PTC Therapeutics, Remix Therapeutics, ReviR Therapeutics, AstraZeneca, Sanofi, Atomic AI, Arrakis Therapeutics, Ribonaut Therapeutics, Ribometrix, and more as they explore the latest advances in RNA target selection, structure-guided design, productive chemical space, screening technologies, and clinical translation.
Meet The Companies Defining The Future Of RNA-Targeted Small Molecules
Connect with the scientists, medicinal chemists, RNA biologists, translational experts, founders, CSOs, and pharma leaders advancing the next generation of RNA-targeted therapeutics.
Built For Teams, Not Individuals
The most successful RNA-targeted drug discovery organizations send teams.
Bring colleagues across:
- RNA Biology
- Drug Discovery
- Medicinal Chemistry
- Structural Biology
- Translational Research
- Business Development
Align your team around the latest advances, emerging modalities, and partnership opportunities shaping the future of RNA-targeted therapeutics.
What Makes This Event Unique?
The Industry's Only Dedicated RNA Small Molecule Meeting
Explore the latest advances exclusively focused on RNA-targeted small molecules, from target discovery through clinical translation.
Learn From The Companies Driving Clinical Progress
Hear the latest scientific insights from the biotech and pharma teams advancing RNA-targeted programs toward therapeutic validation.
Join A Tight-Knit Scientific Community
Build relationships with the researchers, innovators, and decision-makers shaping the future of RNA-targeted therapeutics.
The Questions Defining The Future Of RNA-Targeted Drug Discovery
The industry is approaching an inflection point, but key scientific and translational challenges remain.
Join the leaders tackling questions such as:
How do we move beyond RNA binding to achieve functional modulation?
How can we improve selectivity and reduce off-target effects?
Which RNA targets are truly therapeutically relevant?
Which modalities are most likely to drive the next clinical breakthrough?
Hear how the companies leading the field are addressing these challenges through new data, technologies, and therapeutic strategies.
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
Explore the Scientific Priorities Shaping the Future of RNA Targeting
AI-Driven RNA Targeting
Discover how AI-enabled structure prediction, screening, and target validation are helping researchers identify functionally relevant RNA targets with greater confidence.
Featuring insights from: AstraZeneca, Atomic AI and RevoNA Bio
Expanding RNA Chemical Space
Explore new approaches to RNA-focused chemistry, fragment discovery, and structure-guided design to unlock novel targets beyond traditional chemotypes.
Featuring insights from: University of Colorado Boulder, University of Toronto and Sanofi
Advancing RNA Therapies to the Clinic
Learn how industry leaders are translating RNA-targeting strategies into clinical programs by improving target selection, validation, and therapeutic confidence.
Featuring insights from: PTC Therapeutics, ReviR Therapeutics and Remix Therapeutics.
Driving RNA Therapeutic Outcomes
Discover how innovators are moving beyond RNA binding to achieve meaningful functional modulation and therapeutic impact across multiple disease areas.
Featuring insights from: UNC Chapel Hill, Wayfinder Biosciences, Arrakis Therapeutics, Syrna Therapeutics and Ribometrix.
Future of RNA Small Molecules
Explore emerging modalities including condensate biology, epitranscriptomics, induced proximity, and next-generation RNA-targeting strategies.
Featuring insights from: Dewpoint Therapeutics, EPICS Therapeutics, Sanofi, Pfizer and Ribonaut Therapeutics.
Be Part of the Community Shaping What's Next
RNA-targeted small molecules have never been closer to broader therapeutic validation. As scientific progress accelerates and new programs move into the clinic, collaboration has never been more important.
Join the researchers, innovators, and industry leaders defining the future of RNA-targeted drug discovery.
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.
Partner With Us
Position yourself alongside other leading solution providers to ensure your brand is at the heart of biopharma deals.
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.