Antisense oligonucleotides (ASOs) bind to RNA by Watson-Crick base-pairing and modulate its intermediary metabolism to produce a pharmacological effect. Unmodified oligonucleotides are highly polar, poly-anionic macromolecules which have poor drug-like properties. To address these limitations, medicinal chemists have developed chemical modification and conjugation strategies to improve ASO binding affinity, metabolic stability and pharmacokinetic properties. Recent developments which improve potency and therapeutic efficacy of ASO drugs in animal models will be presented.

Medicinal chemistry strategies for improving the drug-like properties of antisense oligonucleotides

248th National Meeting (2014)

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