RNA Therapy

siRNA-targeted therapy is an emerging therapeutic field after small molecules and antibodies, an outstanding advantage of RNA-targeted therapy is that the identification of drug candidates only needs to determine the target area related to the disease process in RNA, and for any target gene, the corresponding small nucleic acid can be designed to regulate its expression, so RNA therapy has great application potential in traditional undruggable diseases or targets, bringing revolutionary breakthroughs to the research and development of targeted drugs.

Dr. Paul Zamecnik discovered antisense nucleic acids in 1978, pioneering RNA-targeted therapy. RNA-targeted therapy is based on targeted messenger RNA, which regulates the expression of proteins by regulating messenger RNA to treat diseases. Common RNA-targeted therapy techniques include antisense oligonucleotide and small interference RNA, as well as microRNA, small activating RNA and RNA editing. RNA-targeting techniques typically utilize cellular machinery naturally present in the human body, such as RNaseH and RISC, to regulate RNA expression and are relatively safe.

RNA-targeted therapy targets messenger RNA through specific base pairing, which has better specificity and less off-target effects. RNA targeting also has a long drug effect, taking small interfering nucleic acids as an example, it has been able to achieve a dose every 3 to 6 months. At present, 13 oligonucleotide drugs have been approved for marketing worldwide, of which 8 use antisense oligonucleotide technology and 5 are small interfering nucleic acid drugs.