Oligonucleotide therapeutics include small interfering RNA (siRNA), antisense oligonucleotides (ASO), microRNA (miRNA), and aptamers. Composed of nucleotides, oligonucleotide therapeutics represent an entirely new category, distinct from small molecule drugs and antibody drugs, and have several oligonucleotide therapeutics already on the market worldwide. Our main products focus on siRNA therapeutics.
To enable siRNA to selectively target diseased tissues without affecting the physiological functions of normal tissues, delivery technologies are required. siRNA modification technologies further allow for lower effective doses and long duration. We have been dedicated to innovation in siRNA delivery and modification technologies, establishing multiple proprietary technology platforms.
siRNA
siRNA is a double-stranded short RNA molecule capable of binding to AGO proteins to form an RNA-induced silencing complex (RISC). In this process, one strand (Passenger Strand) is degraded, while the other (Guide Strand) binds to and degrades target mRNA through complementary base pairing, thereby silencing target genes and inhibiting the expression of disease-related proteins. This mechanism, known as RNA interference (RNAi), was awarded the 2006 Nobel Prize in Physiology or Medicine. RNAi garnered significant scientific acclaim, named a top ten breakthrough in 2001 and 2002 by "Science" and "Nature," respectively.
