Table 1.
Name | ASO | Target exon | Phase | ||
---|---|---|---|---|---|
Nucleic acid | Size | Route | |||
Eteplirsen | PMO | 25 | IV | 51 | Approved |
Golodirsen | PMO | 25 | IV | 53 | Approved |
Viltolarsen | PMO | 21 | IV | 53 | Approved |
Casimersen | PMO | 25 | IV | 45 | Approval stage |
Renadirsen | ENA/2'RNA | 18 | SC | 45 | I/II |
PMO: phosphorodiamidate morpholino; ENA: 2'-O,4'-C-ethylene-bridged nucleic acid; 2'RNA: 2'-O-methyl RNA; IV: intravenous injection; SC: subcutaneous injection