The 9th Annual RNA-Targeted Drug Discovery & Development Summit unites experts to advance the discovery, design, and development of RNA-targeted therapeutics across diverse disease areas through innovative science and strategic collaboration.
Key Topics
Targeting pathological RNA in myotonic dystrophy with small molecules
Emerging modalities beyond splice modulation, including covalent binders and lncRNA targets
Advancing RNA-targeted therapeutics for translation across disease areas
Harnessing RNA splice modulators for nucleotide repeat disorders
Breaking dimensional limits in RNA–ligand discovery
Large-scale discovery of RNA tertiary structures and disease-relevant targets
Drugging RNA–protein complexes with small molecules
Advancing epitranscriptomics with METTL3 inhibitors
Expanding chemical and mechanistic diversity for orally available RNA therapeutics
Context-dependent translation inhibition for cancer therapy
Targeting HTT RNA for allele-selective Huntington’s treatment
Optimizing chemical matter for improved RNA therapeutics
Strategic collaborations and investment in RNA-targeted drug discovery
Targeting structured RNA motifs for selective functional binders
RNA target selection and functional binder design
Fungal RNA and peptide-ribosome complexes as therapeutic targets
Targeting group I introns for novel antifungal mechanisms
Splicing modulation therapies for neurodegenerative diseases
Conformation-driven screening to discover RNA binders
Small molecule modulation of MALAT1 substructures
PTC splice modulators as a therapeutic platform
Who should Attend
Medicinal chemists and structural biologists
Pharmaceutical and biotech research leaders
RNA biology and therapeutics researchers
Business development and investment professionals
Drug discovery scientists in oncology, neurology, and rare diseases
Translational medicine specialists
Platform technology innovators
Venue
Hotel Commonwealth, 500 Commonwealth Avenue, Boston, Massachusetts, United States
