Table 5.
A spectrum of clinical trials of siRNA-based drugs.
| Commercial Name | Substance | Clinical Trial No. | Target | Progress |
|---|---|---|---|---|
| ONPATTRO | patisiran |
NCT01617967 NCT02510261 NCT04201418 NCT03997383 NCT05040373 |
Transthyretin (TTR) | FDA approved, long-term studies, pregnancy safety studies |
| LEQVIO | Inclisiran [92] |
NCT05362903, NCT04929249 NCT05682378 NCT03159416 NCT05399992 |
Proprotein convertase subtilisin/kexin type 9 (PCSK9) | FDA approved, long-term study, combination therapy effectiveness study, extension trials |
| OXLUMO | Lumasiran |
NCT04152200 NCT03905694 NCT03350451 NCT04982393 |
Hydroxyacid oxidase (OA1) | FDA approved, observational study, extension trials |
| GIVLAARI | givosiran |
NCT04883905 NCT02452372 |
aminolevulinic acid synthase 1 (ALAS1) | FDA approved, combination therapy |
| - | Cemdisiran |
NCT02352493 NCT05070858 NCT04601844 NCT05744921 NCT05133531 |
Complement 5 | Phase I/II completed, Combination therapy trials |
| Anti-EpHa2 siRNA | Anti-EpHa2 siRNA | NCT01591356 | ephrin type-A receptor 2 (EpHa2) | Phase I estimated in 2024 |
| CAS3/SS3 | Anti-CpG-STAT3 siRNA | NCT04995536 | TLR9 receptor and signal transducer and activator of transcription 3 (STAT3) | Phase I estimated in 2024 |
| NBF-006 | Anti-KRAS siRNA | NCT03819387 | KRAS proto-oncogene (KRAS) | Phase I estimated in 2024 |
| ALN-KHK | antiKHK siRNA | NCT05761301 | ketohexokinase (KHK) | Phase I/II estimated in 2025 |
| OLX10212 | Asymmetric siRNA | NCT05643118 | Pathways upstream of VEGF (vascular endothelial growth factor) | Phase I estimated in 2024 |
| ADX-038 | Anti-PK siRNA | NCT05876312 | Prekallikrein (PK), | Phase I estimated in 2025 |
| SRN-001 | Anti-AREG siRNA | NCT05984992 | Amphiregulin (AREG) | Phase I estimated in 2024 |
| AOC 1020 | Anti-DUX4 siRNA | NCT05747924 | Double homeobox 4(DUX4) | Phase I estimated in 2025 |
| AGX148/PH-762 | Anti PD-1 siRNA | NCT05902520 | siRNA Modulation of PD-1 | Phase I estimated in 2026 |