FDA Approved Oligonucleotide Drugs

Synthetic oligonucleotides are the key factor in almost all molecular biological methodologies. They are used in PCR, reverse transcription, sequencing, gene editing, synthetic biology, cloning, single nucleotide polymorphism analysis, microarray, electrophoretic mobility analysis, and small RNA studies. Most of these technological advances require synthesized oligonucleotides of high quality, completeness, and purity.

The currently marketed oligonucleotide drugs are mainly chemically modified to improve the metabolic stability of nucleotide structure and reduce adverse reactions, for instance, the modification of phosphodiester bonds, ribose, nucleotide bases, or ribose-phosphate backbones. Compared with the traditional drug development model, the new generation oligonucleotide drugs have obvious advantages. Conventional small-molecule drug development requires a great deal of screening and optimization process while oligonucleotide drug candidates can be quickly screened based on the primary gene sequence, and then high-specificity lead compounds can be designed. In addition, the unique function mechanism of nucleotide drugs allows them to modulate target activities that are not recognized by small molecules, thus providing a wider range of clinical applications.

Classifications and research focus

<strong><a href="https://www.bocsci.com/blog/fda-approved-oligonucleotide-drugs/">Those the antisense oligonucleotides drugs</a></strong>, small interfering ribonucleic acid (siRNA), ribozyme, deoxyribozyme, etc. are all concluded as nucleotide drugs. Oligonucleotides can regulate gene expression through a series of processes such as RNA interference, ribonuclease-mediated degradation, splicing regulation, non-coding RNA inhibition, gene activation, and programmed genome editing. ASO and siRNA are recognized as the most commonly used gene regulation tools, which are widely used and have been developed as gene therapy drugs.

FDA approval

Over 20 years of research and clinical trials, 14 small nucleic acid-based drugs have entered the market by 2021. The FDA-approved oligonucleotide drugs are listed in the table below.

The above drugs are mostly used for genetic and rare diseases. At present, there are still a large number of oligonucleotide drugs in the research and clinical stage, indicating great prospects for future development.

BOC Sciences perspective

BOC Sciences also deems that oligonucleotide drugs have a broad market prospect. As a new category of gene therapy drugs, oligonucleotide drugs have been rapidly developed, providing hope for many rare or incurable diseases. However, the process of oligonucleotide drugs is complicated and it’s difficult to carry out impurity analysis. BOC Sciences will quicken its pace of innovation, aiming to provide high-quality nucleotide materials and custom synthesis support.