TargetRNA is a biotechnology company that discovers novel small molecule drugs that act directly on disease-causing RNA.

Our discovery platform uniquely integrates high throughput biophysics, machine learning, NMR spectroscopy, and molecular modeling to search previously unexplored chemical diversity space for fragments that bind to discrete pockets in folded RNA structures.

Computational and medicinal chemistry optimization cycles are guided by atomic-level structural information to design selective, high affinity lead molecules.  This increases the success rate of leads in phenotypic, cell-based assays and pre-clinical animal models.