CMC: Regulatory landscape

CMC (chemistry, manufacturing and control) for oligonucleotide therapeutics, synthesized on solid phase synthesis and purified by column chromatography—the most common way to manufacture such molecules at the moment—is an unusual regulatory situation compared to small molecules and even to synthetic peptide due to the relative short experience and the various types of oligonucleotide. Some of the usual ICH guidelines for drug substances and drug products do not fully apply. This chapter will provide some suggestions on what can be done based on the author’s experience.