TABLE 3.
Clinical trials using stop codon read-through and exon skipping for DMD therapy in 2023.
| Drug name | Description | Company | Delivery route | Current stage | Clinical trial number |
|---|---|---|---|---|---|
| Stop codon read-through | |||||
| Ataluren | Nonsense suppression | PTC Therapeutics | Oral | Phase III | NCT01557400 |
| Exon skipping | |||||
| Eteplirsen (AVI-4568) | PMO morpholino targeting exon 51 | Sarepta Therapeutics | Intravenous | Phase II | NCT03218995 |
| Phase II | NCT04179409 | ||||
| Phase III | NCT03992430 | ||||
| Phase III | NCT03985878 | ||||
| Golodirsen (SRP4053) | PMO morpholino targeting exon 53 | Sarepta Therapeutics | Intravenous | Phase II | NCT04179409 |
| Phase III | NCT02500381 | ||||
| Phase III | NCT03532542 | ||||
| Casimersen (SRP4053) | PMO morpholino targeting exon 45 | Sarepta Therapeutics | Intravenous | Phase II | NCT04179409 |
| Viltolarsen (NCNP-01) | PMO morpholino targeting exon 53 | Nippon Shinyaku Co. Ltd | Intravenous | Phase II | NCT03167255 |
| Drisapersen (PRO051) | 2′-O-methyl PS Targeting exon 51 | BioMarin Pharmaceutical Inc | Intravenous | Extension | NCT02636686 |