September_New Drugs|Nucleic Acids: A New Blue Ocean: New Drug Development Opportunities in the Spotlight
Nucleic acid therapy has unlimited potential and has become the focus of technology, capital and market pursuit
Nucleic acid therapy is a series of nucleotides composed of drugs, for the treatment of human genetic diseases, rare diseases, tumors, chronic diseases, and infectious diseases to provide new treatment options, is expected to succeed the small molecule chemical drugs and protein antagonists as a new class (modality) of mainstream drugs. Proteins (Remarks: Proteins are gene products) are important components of life activities and diseases, and their mechanism of action is closely related to diseases and is also the core of drug development. According to the analysis of Nature Reviews journal, due to the structural characteristics of proteins, there are about 85% target disease-causing proteins or disease-associated proteins that cannot be acted upon by small molecule chemicals or protein antiseptics, and therefore cannot be made into drugs. The core value of nucleic acid drugs is that they can break through the difficulties in protein structure design and selectively inhibit the disease-causing proteins associated with the undruggable protein targets at the molecular level by inhibiting gene expression, targeting non-coding RNAs involved in transcriptional and epigenetic regulation, changing mRNA splicing, up-regulating the target genes and editing genes, etc., so as to carry out the treatment of the relevant diseases, which theoretically can be directed against a wide range of disease-causing proteins. Theoretically, it can inhibit a wide range of disease-causing proteins, and therefore new nucleic acid drugs are attracting attention in the market.