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.