Nucleic Acid-Based Drugs

Nucleic Acid-Based Drugs

Most drugs available today work by interacting with certain proteins, which are the cause of disease. However, they often have side effects, as they also interact with non-target proteins.
Nucleic acid therapeutics, based on nucleic acids or closely related chemical compounds, are an emerging new class of therapeutics for treating unmet medical needs. They are capable of targeting a disease at the genetic level by preventing the expression of disease-causing proteins.


These nucleic acid drugs can be divided into six categories based on their structures, targets, and mechanisms of action.

Category Structure Target Mechanism
Antisense Single-strand DNA/RNA ・mRNA
・Cleavage mRNA(RNase-H)
・Exon skipping
・Cleavage miRNA
siRNA Double-strand RNA
(hair-pine type is Single-strand)
mRNA Cleavage mRNA
(RNA interference)
miRNA Double-strand RNA
(Pre-miRNA is Single-strand)
mRNA miRNA replacement
(RNA interference)
Aptamer Single-strand DNA/RNA Protein
(Outside of cells)
Inhibits the physiological
Decoy Double-strand DNA Transcription factor Inhibits transcription
CpG-oligo Single-strand DNA TLR9-receptor adjuvant
  1. 1. Antisense oligonucleotides, siRNA, and miRNA consist of 15 to 25 nucleotides, which are complementary or partially complementary to their messenger RNA and down-regulate translation of target proteins.
  2. 2. Aptamers can recognize target proteins by their molecular structure and interact directly with the target proteins.
  3. 3. Decoy and CpG-oligo are designed to inhibit the activity of the target protein through high-affinity binding.

The active ingredients in nucleic acid drugs are oligonucleotides, mainly produced through chemical synthesis, like traditional small molecules. Sumitomo Chemical is a leading Japanese manufacturer of small molecule active pharmaceutical ingredients (APIs) and advanced intermediates for supply to the global pharmaceutical industry.
With our expertise in small molecule manufacturing, state-of-the-art technology platform, sophisticated QA system, and experience in regulatory services, we provide the best possible service to our customers as a supplier of oligonucleotide APIs.