Synthetic oligonucleotides are short nucleic acid chains that can control gene expression in a sequence-specific manner. They can target different diseases by interfering with RNA functions at the cellular level. As a new class of therapeutics, synthetic oligonucleotides have shown great promise in the treatment of neurological disorders, metabolic diseases, cancers, and other diseases.
There is limited published guidance from regulatory agencies on quality control of oligonucleotide drugs, including control of raw materials or product-related impurities. USP now offers standards to support quality assessment of critical raw materials used in oligonucleotide synthesis. These standards also help you navigate the current regulatory environment around assessing product-related impurities that can affect safety and efficacy.