RNA-Targeted Small Molecules: From Discovery to Clinic

RNA-targeted small molecules are rapidly emerging as one of the most exciting areas in drug discovery, with growing clinical validation demonstrating the potential to address historically undruggable targets across oncology, rare genetic diseases, and neurodegenerative disorders.

This November, leading scientists from across pharma, biotech, and academia will gather in Boston for the 9th RNA-Targeted Drug Discovery & Development Summit, the industry's dedicated forum for advancing RNA-targeted small molecule therapeutics.

As the field advances beyond traditional splice modulation strategies, researchers are exploring a broader range of approaches including direct RNA binding, RNA degradation, covalent translation inhibition, epitranscriptomic regulation, and condensate biology. These innovations are creating new opportunities to target disease-driving RNA structures and expand the therapeutic landscape and will get explored at the conference.

The meeting will feature experts from organizations including AstraZeneca, Sanofi, Pfizer, PTC Therapeutics, Remix Therapeutics, ReviR Therapeutics, Arrakis Therapeutics, Atomic AI, Dewpoint Therapeutics, EPICS Therapeutics, Ribonaut Therapeutics, Wayfinder Biosciences, Syrna Therapeutics, and more.

Attendees will gain practical insights into some of the most pressing challenges facing RNA-targeted drug discovery, including:

• Identifying functionally relevant and tractable RNA targets
• Leveraging AI and computational biology for RNA structure prediction and target selection
• Expanding RNA-focused chemical space beyond established chemotypes
• Advancing direct RNA-binding approaches and RNA degraders
• Improving selectivity, target engagement, and translational confidence
• Progressing RNA-targeted therapeutics toward clinical development

Download the Event Guide to learn more here.

The summit is designed for a broad cross-section of the RNA therapeutics community.

1. Large pharma leaders can benchmark emerging RNA-targeting strategies and evaluate the modality's growing therapeutic potential.
2. Biotech executives and drug developers can learn how industry pioneers are advancing RNA-targeted pipelines, overcoming discovery challenges, and generating clinical momentum.
3. Discovery and translational scientists working in RNA biology, medicinal chemistry, structural biology, computational biology, screening, and drug discovery will gain actionable scientific insights that can be applied directly to ongoing programmes.
4. Academic researchers will have the opportunity to engage with industry leaders and explore the latest advances in RNA structure, biology, and druggability.

View why you should attend here.

As investment, innovation, and clinical progress continue to accelerate, RNA-targeted therapeutics are moving from scientific promise toward therapeutic reality. For organizations looking to stay at the forefront of RNA drug discovery, this summit offers a unique opportunity to learn from the companies and researchers shaping the future of the field.

We have recently seen groups from Skyhawk Therapeutics, Sanofi, Bayer, PTC Therapeutics, Chugai Pharmaceuticals, Nippon Shinyaku and others book on. Join the RNA-Targeted Small Molecule community in Boston this November:

Register to Attend & Save.