Table 1.
RNA-based drugs and biomarkers for cardiac and skeletal pathologies.
| Drug | RNA Type | Target | Disease/Condition | Company | Phase | Reference |
|---|---|---|---|---|---|---|
| MRG-110 | Anti-miR | miR-92a | Wound Healing | miRagen (Viridian) |
Phase I | NCT03603431 |
| Spinraza (Nusinersen) |
ASO | SMN2 | SMA | Ionis | FDA/EMA approved |
NDA:209531 EMEA/H/C/004312 |
| Eteplirsen (Exondys 51) |
ASO | Dystrophin | DMD | Sarepta Inotersen |
FDA approved |
NDA:206488 |
| Drisapersen (Kyndrisa) |
ASO | Dystrophin | DMD | BioMarin | Phase III | NCT02636686 |
| Vitolarsen (Viltepso) |
ASO | Dystrophin | DMD | Nippon Shinyaku | FDA approved |
NDA:212154 |
| Golodirsen (Vyondis 53) |
ASO | Dystrophin | DMD | Sarepta Therapeutics |
FDA approved |
NDA:211970 |
| Casimersen (Amondys 45) |
ASO | Dystrophin | DMD | Sarepta Therapeutics |
FDA approved |
NDA:213026 |
| TQJ230 | siRNA | Apo(a) | Cardiovascular Disease, Elevated Lp(a) |
Novartis | Phase III | NCT04023552 |
| AZD8601 | mRNA | VEGF | Ischemic Heart Disease | Moderna, Astrazeneca |
Phase II | NCT03370887 |
| HEARTBiT | miR | Biomarker | Heart Transplant Rejection |
NCT03575910 | ||
| CRUCIAL | Circulating RNAs | Biomarker | Acute Heart Failure | NCT03345446 |