Research Overview

RNA therapeutics is a rapidly expanding area of next-generation biological drugs. RNA is a versatile molecule and naturally regulates gene expression, either by upregulation or downregulation. This function is employed to design novel RNA therapeutics. The fundamentals of RNA therapeutics consists of delivering functional RNA at a target; however, this is restricted due to its fragile nature and lack of appropriate delivery systems.

Advances in RNA stability, chemical modification, and delivery systems have led to the commercial translation of RNA therapeutics during the past two decades. Moreover, RNAs are relatively easy to manufacture, are cost-effective, and can act on undruggable targets. Conventionally, only academic labs and small biotech companies focus on RNA therapeutics research.

Previously, RNA therapeutics R&D was focused on rare diseases and cancer. However, recent advances in chemical modification, improved stability, formulation, and nucleic acid delivery systems have improved the prospects of RNA therapeutics, and pharmaceutical and biotechnology interest are on the rise.