Nucleic acids—including DNA and RNA—are biological macromolecules responsible for carrying, encoding, and transmitting genetic information within cells. Beyond serving as genetic information carriers, they play decisive roles in growth, development, heredity, and variation through transcription/reverse transcription and translation.

Traditional drugs target proteins. However, many "disease-causing proteins" are undruggable targets, leaving many diseases without effective treatments. With increasing disease complexity, only gene-level therapy offers permanent solutions. This is where nucleic acid drugs—using nucleic acid molecules for translation or regulatory functions—come in.

What Are Nucleic Acid Drugs?

Nucleic acid drugs are compounds containing nucleotide or deoxynucleotide structures with genetic characteristics and pharmacological activity. They act on pathogenic target genes or target mRNAs, regulating gene expression at the source to prevent or treat disease. Typically 12–30 nucleotides in length, they are also called oligonucleotide drugs, including RNA drugs (ASO, siRNA, miRNA, saRNA, mRNA, RNA aptamers, ribozymes, antibody-nucleic acid conjugates) and DNA drugs.

As a form of gene therapy, nucleic acid drugs represent the next generation of therapeutics after small molecules and antibodies. They act at the gene level, directly targeting disease-causing molecules without manipulating the genome—offering both symptomatic and root-cause treatment.

Since the first nucleic acid drug (fomivirsen sodium) was approved in 1998, dozens have been approved globally. Nucleic acid drugs show tremendous potential with advantages including rich targets, high specificity, high efficiency, low toxicity, and significant applications in antivirals, oncology, and metabolic disorders. However, challenges remain—nucleic acid specificity, stability, and delivery efficiency are key factors for efficacy. With advancing molecular biology and delivery technologies, nucleic acid drugs are among the most promising areas in biopharmaceutical R&D.