Antisense Oligonucleotides are the Rising Star of RNA-targeted Drugs

From the end of the 19th century to present, the global biopharmaceutical industry has been continuously developing and innovating. During this time, the industry has experienced the industrial wave of small molecule compounds, recombinant antibodies drugs, and RNA-targeted drugs. Studies have revealed that more than 80% of disease-related proteins are not available as drug targets by conventional small molecule drugs or biomacromolecule formulations, However, RNA-targeted drugs play a role at the transcriptional level, which can greatly expand the proportion of therapeutic targets in the human genome. RNA-targeting drugs mainly include antisense oligonucleotides (ASOs), small interfering RNA (siRNA), microRNA (miRNA), messenger RNA (mRNA), RNA Aptamers (Aptamers), et al. With the progress of antisense technology, including RNA delivery technology, chemical modification, pharmacodynamic studies and so on, RNA-targeted drug development platforms continue to mature. Currently, antisense oligonucleotides (ASOs) and small interfering RNA (siRNA) are the main forms of RNA-targeted drug development. The ten commercially available RNA-targeted drugs include eight single-stranded antisense drugs (ASOs) and two double-stranded antisense drugs (siRNA).

According to the principle of Watson–Crick base pairs, the designed antisense nucleic acid can specifically bind to the target mRNA sequence or ncRNA sequence and regulate gene expression at the transcriptional level. Antisense RNA can change the structure of mature RNA by affecting the splicing, processing, and modification of precursor RNA, or affect the metabolism of RNA through degradation and make it unable to be translated into proteins.

Antisense oligonucleotides (ASOs) are a kind of short, single chain oligonucleotide that can consist of DNA, RNA, or DNA/RNA heterozygous sequences. The common antisense mechanism includes the use of endoribonuclease RNase H1. When ASOs bind to the target RNA, RNase H1 will cleave the target RNA sequence, leading to the degradation of it. While, in order to enter the cell, ASOs must be modified to avoid the degradation of nuclease. At present, familiar modifications include backbone modifications, such as phosphorothioate (PS) backbone modifications, and 2′-ribose modifications, such as 2′-O-methy (OMe), 2′-O-methoxyethyl (MOE), locked nucleic acid (LNA), or 2′-fluoro (F). These modifications can increase the stability, cell uptake of ASOs, and improve the overall performance of ASOs.

With nearly twenty years of experience in oligonucleotide synthesis, Synbio Technologies can provide trusted professional technical support and production output of ASOs for our customers. Various modifications, flexible synthesis scale, and fast delivery turnaround time keep us competitive in the international markets. We are pleased to provide you with high quality ASOs products.

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