RNA therapeutics refers to a class of medicine that uses ribonucleic acid (RNA)—a molecule that plays a critical role in the coding, decoding, regulation, and expression of genes—to treat diseases. Unlike traditional drugs that target proteins, this approach targets the underlying genetic instructions at the RNA level to deliver more precise and effective treatments for various conditions including genetic disorders, cancer, and infectious diseases. This rapidly evolving practice plays a significant role in advancing biotechnology, medicine, and research and, in particular, has fostered the development of precision medicine.

The RNA therapeutics industry comprises companies that focus on developing treatments based on RNA molecules. These companies can be broadly categorized as a) those providing endogenous RNA targeting (i.e., ASOs/PMOs, RNAi), b) those developing other RNA molecules (i.e., tRNA, circRNA, IncRNA, etc.), and c) those providing RNA sequence and structure modulation.

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