Direct RNA Binding Emerges as a Powerful Approach for Functional Disease Modulation

While splice modulation has produced some of the field’s most significant successes to date, researchers are increasingly exploring direct RNA binding as a route to achieving therapeutic outcomes across a broader range of diseases.

The upcoming RNA-Targeted Drug Discovery & Development Summit will feature multiple presentations demonstrating how direct RNA-targeting strategies are being translated into functional biological effects.

Among the featured sessions, Kevin Weeks of the University of North Carolina at Chapel Hill will present a framework for discovering direct RNA-targeting molecules capable of achieving translation knockdown through fragment-based approaches.

David Sparkman-Yager, Chief Technology Officer at Wayfinder Biosciences, will discuss the optimization and functional characterization of MYC RNA-targeting small molecules, including work to enhance potency, selectivity, and in vivo efficacy.

Additionally, Domi Stickens, Chief Scientific Officer at Arrakis Therapeutics, will highlight efforts to restore splicing function in myotonic dystrophy type 1 (DM1) through selective targeting of toxic CUG-repeat RNA.

As researchers seek to access previously undruggable disease drivers, direct RNA binding is increasingly viewed as one of the most exciting frontiers in small molecule drug discovery.

View the 2026 event guide to see why this year’s attendance is essential.