A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides.

ABSTRACT

Oligonucleotide therapeutics, a pioneering category of modern medicinal drugs, are at the forefront of utilizing innate mechanisms to modulate gene expression. With 18 oligonucleotide-based FDA-approved medicines currently available for treating various clinical conditions, this field showcases an innovative potential yet to be fully explored. Factors such as purity, formulation, and endotoxin levels profoundly influence the efficacy and safety of these therapeutics. Therefore, a thorough understanding of the chemical factors essential for producing high-quality oligonucleotides for preclinical studies is crucial in their development for further clinical application. This paper serves as a concise guide to these chemical considerations, aiming to inspire and equip researchers with the necessary knowledge to advance in this exciting and innovative field.