Optimizing RNA-Targeting Small Molecules Through Structure-Guided Design, Selective Modulation, and Translational Strategies to Develop Safe, Bioavailable, and Clinically Effective Therapies

RNA-targeting small molecules are rapidly emerging as a powerful therapeutic reality. From modulating splicing to directly binding RNAs, these molecules offer a revolutionary approach to treating previously undruggable diseases across neurodegenerative, cancer and rare disorders. In 2025 alone, the field has seen major collaborations, including Wayfinder Biosciences with Daiichi Sankyo, Ono Pharmaceutical with Reborna Biosciences, and xFOREST with Astellas Pharma—signaling growing momentum and industry confidence.

Now in its 8th year, the RNA-Targeted Drug Discovery & Development Summit returns as the only dedicated industry forum addressing the unique challenges of RNA-targeted drug development across splice modulators, covalent drugs, proximity inducers and direct RNA binders.

Join 25+ world-class speakers from leaders like PTC Therapeutics, ReviR Therapeutics, Remix Therapeutics, Rgenta Therapeutics, Arrakis Therapeutics and more this December to gain the connections, tools and scientific firepower needed to bring your RNA-targeted therapeutics to clinic and beyond.