Potential for small molecule drug discovery targeting RNA

Abstract

Drug discovery research with protein-targeted small molecules is becoming difficult because proteins as potential drug targets are already exhausted. For this reason, RNA, which is responsible for protein synthesis, is attracting attention from pharmaceutical companies as new target for drug discovery. Since RNA is essential for the modulation of genetic information and gene regulation in human cells and for maintaining biological functions, it has been shown to be directly involved in many genetic diseases. However, unlike proteins, RNA molecules contain structured regions and are inherently flexible, and their conformational changes have been reported to play an important role in maintaining biological functions. In order to validate RNAs with these characteristics as new drug target to replace proteins, drug discovery research should be conducted from the perspective of chemistry, molecular biology and pharmacology. In this section, we will outline the examples of RNA-targeted small molecules under clinical development and characteristics of them, as well as novel approach in Reborna Biosciences, Inc.