Biomolecules & Therapeutics : eISSN 2005-4483 / pISSN 1976-9148

Table. 1.

Table 1 Approved ASO drugs

Drug Approval year Target indication Target gene Mode of action Chemistry
Formivirsen 1998 CMV retinitis CMV RNase H1 PS-ODN
Mipomersen 2013 HoFH ApoB-100 RNase H1 PS-MOE gapmer
Nusinersen 2016 SMA SMN2 intron 7 Exon inclusion PS-MOE
Eteplirsen 2016 DMD Dystrophin exon 51 Exon skipping PMO
Inotersen 2018 hATTR TTR RNase H1 PS-MOE gapmer
Golodirsen 2019 DMD Dystrophin exon 53 Exon skipping PMO
Volanesorsen 2019 FCS ApoC-III RNase H1 PS-MOE gapmer
Viltolarsen 2020 DMD Dystrophin exon 53 Exon skipping PMO
Casimersen 2021 DMD Dystrophin exon 45 Exon skipping PMO

ApoB-100, apolipoprotein B-100; ApoC-III, apolipoprotein C-III; CMV, cytomegalovirus; DMD, Duchenne muscular dystrophy; hATTR, hereditary transthyretin-mediated amyloidosis; FCS, familial chylomicronemia syndrome; HoFH, homozygous familial hypercholesterolemia; MOE, 2′-methoxyethyl; ODN, oligodeoxynucleotide; PMO, phosphorodiamidate morpholino oligonucleotide; PS, phosphorothioate; SMA, spinal muscular atrophy; TTR, transthyretin. For details on chemistries, refer to the figures below.

Biomolecules & Therapeutics 2023;31:241~252 https://doi.org/10.4062/biomolther.2023.001
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