What are the advantages of Oligonucleotide drugs?

The emergence of oligonucleotide drugs makes it possible to actively design drug sequences to target and silence disease genes, which has multiple advantages compared with traditional drugs.

• Rich candidate targets and wide distribution of indications

Theoretically, any disease caused by the overexpression of a specific gene can be treated with oligonucleotide drugs, which provides a wealth of candidate targets for the development of oligonucleotide drugs, including many targets that cannot be cured by traditional drugs.

• Easy drug design and short development cycle

Oligonucleic acid drugs are the area that has benefited the most from the development of genomics and functional genomics so far. The design of oligonucleotide drugs can realize digital design directly targeting disease gene (mRNA) sequences, thereby getting rid of the large-scale screening cycle of traditional drugs, allowing drug design to be proactively carried out, and the success rate of research and development is relatively high.

• Strong targeting specificity

Oligonucleic acid drugs regulate gene expression from the post-transcriptional level, can specifically act on the mRNA transcribed by disease-causing genes, regulate the expression of disease-causing genes from the upstream of the disease, and can achieve sequence specificity at the single-base level. It has the characteristics of "targeting the target" and "treating the symptoms and root cause". In addition, nucleic acid drug delivery systems are constantly innovating, which can effectively deliver nucleic acid drugs to target organs and truly achieve targeted drug delivery.

• Long-lasting drug action

Oligonucleic acid drugs target mRNA. Taking siRNA drugs as an example, when the target mRNA is degraded, the RNA-induced silencing complex can work in a cycle to participate in the next round of degradation of the target mRNA. Therefore, oligonucleotide drugs can achieve longer-term effects in cells. In addition, the chemical modification technology of nucleic acid drugs continues to innovate, including phosphate backbone modification, sugar modification and base modification, etc., which can improve the stability and half-life of nucleic acid drugs, make the drug more effective, and greatly improve the compliance of patients with treatment.