Oligonucleotides

Oligonucleotides are short nucleic acid polymers with defined sequences, encompassing classes such as antisense oligonucleotides (ASOs), small interfering RNA (siRNA), microRNA (miRNA), and aptamers. These molecules are capable of modulating gene expression through diverse mechanisms, including RNA interference (RNAi), RNase H-mediated target RNA degradation, splice modulation, inhibition of non-coding RNAs, gene activation, and programmable genome editing.A key advantage of oligonucleotides lies in their ability to hybridize with target mRNA or pre-mRNA via complementary base pairing. This property enables highly selective regulation of gene and protein expression, allowing, in principle, the targeting of virtually any gene. Consequently, oligonucleotide-based therapeutics represent a fundamentally distinct approach compared to conventional small-molecule inhibitors. Their potential in precision medicine has generated significant interest, particularly in the treatment of cancer, cardiovascular disorders, and rare genetic diseases. Notably, recent regulatory approvals of agents such as Givosiran(T39123),Lumasiran(T39273),Viltolarsen(T39455) have accelerated the integration of RNA-based therapies into mainstream drug development.