Platform Strategies for Synthetic Oligonucleotide Drug Substances

Abstract

Synthetic therapeutic oligonucleotide drug substances are commonly manufactured using the same basic unit operations, i.e., solid phase oligonucleotide synthesis, cleavage and deprotection, purification, and isolation. The platform nature of oligonucleotide drug substance manufacturing provides opportunities to optimize process development, streamline process validation, and expedite regulatory submissions. In this publication, we discuss a modular approach to platform strategies for synthetic oligonucleotide drug substances, the opportunities associated with the application of platform knowledge, and how such information could be presented in regulatory filings.