Table 2.
siRNA-based drugs in clinical trials (As of 1st May 2023, surveys are available at clinicaltrials.gov.).
| Drugs | First posted | Target | Delivery system | Cancer type | Status | Company | ClinicalTrials.gov identifier |
|---|---|---|---|---|---|---|---|
| SY | 2018–08–01 | KRAS G12D | Exosomes | Metastatic Pancreatic; Adenocarcinoma Pancreatic Ductal Adenocarcinoma Stage IV; Pancreatic Cancer AJCC v8 | Phase 1 Recruiting | M.D. Anderson Cancer Center | NCT03608631 |
| siRNA-EphA2-DOPC | 2012–05–04 | EphA2 | LNP | Advanced cancers | Phase 1 Recruiting | M.D. Anderson Cancer Center | NCT01591356 |
| Atu027 | 2009–07–14 | PKN3 | LNP | Solid tumours | Phase 1 Completed | Silence Therapeutics | NCT00938574 |
| CALAA-01 | 2008–06–03 | RRM2 | Cyclodextrin NP | Solid tumours | Phase 1 Terminated | Calando Pharmaceuticals | NCT00689065 |
| Unknown | 2008–05–06 | Proteasome | Naked siRNA | Metastatic Melanoma | Phase 1 Completed | Scott Pruitt | NCT00672542 |
| APN401 | 2014–06–18 | siRNA-transfected | Naked siRNA | Solid Tumors | Phase 1 Completed | Wake Forest University Health Sciences | NCT0216625 |
| APN401 | 2017–03–22 | siRNA-transfected | Naked siRNA | Solid Tumors | Phase 1 Completed | Wake Forest University Health Sciences | NCT03087591 |
| PSCT19 | 2015–08–19 | PD-L1/PD-L2 | Naked siRNA | Hematological Malignancies | Phase 2 Completed | Radboud University Medical Center | NCT02528682 |
| siG12D LODER | 2012–08–30 | KRAS | LODER polymer | Pancreatic Ductal AdenocarcinomaPancreatic Cancer | Phase 2 Recruiting | Silenseed | NCT01676259 |
| SXL01 | 2016–08–15 | androgen receptor | Naked siRNA | Prostatic Cancer | Phase 1 Withdrawn | Institut Claudius Regaud | NCT02866916 |
| Unknown | 2005–11–23 | Unknown | SV40 vectors | Chronic Myeloid Leukemia | Completed | Hadassah Medical Organization | NCT00257647 |
| CAS3/SS3 | 2021–08–09 | TLR9 receptor; STAT3 | CpG conjugated | Relapsed/Refractory B-Cell NHL | Phase 1 Recruiting | City of Hope Medical Center | NCT04995536 |
| Unknown | 2011–01–05 | BCL-B | Unknown | Multiple Myeloma | unknown | Center hospitalier universitaire de nice | NCT01270009 |
| STP705 | 2021–04–14 | TGF-β1; COX-2 | Unknown | Squamous Cell Carcinoma in Situ | Phase 2 Recruiting | Sirnaomics | NCT04844983 |
| Atu027 | 2013–03–11 | PKN3 | Cationic lipoplex | Carcinoma, Pancreatic Ductal | Phase 1/2 Completed | Silence Therapeutics | NCT01808638 |
| Unknown | 2010–02–25 | E2F1; MYCN | Unknown | Neuroblastoma | Completed | National Taiwan University Hospital | NCT01075360 |
| Unknown | 2010–01–29 | B4GALNT3 | Unknown | Neuroblastoma | Completed | National Taiwan University Hospital | NCT01058798 |
| SLN124 | 2019–11–25 | TMPRSS6 | GalNAc conjugated | Beta-thalassaemia; Myelodysplastic Syndrome | Phase 1 Withdrawn | Silence Therapeutics | NCT04176653 |
| SLN124 | 2022–08–12 | TMPRSS6 | GalNAc conjugated | Polycythemia Vera | Phase 1/2 Not yet recruiting | Silence Therapeutics | NCT05499013 |
| NU-0129 | 2017–01–13 | BCL-2 L12 | AuNPs | Gliosarcoma | Phase 1 Completed | Northwestern University | NCT03020017 |
| NBF-006 | 2019–01–28 | GSTP | LNP | NSCLC; Pancreatic Cancer; CRC | Phase 1 Recruiting | Nitto BioPharma | NCT03819387 |
| STP705 | 2021–04–14 | TGF-β1; COX-2 | Copolymer PNPs | Keloid | Phase 2 Recruiting | Sirnaomics | NCT04844840 |
| ALN-VSP02 | 2010–07–08 | KSP; VEGF | SNALP | Solid Tumors | Phase 1 Completed | Alnylam Pharmaceuticals | NCT01158079 |
| TKM-080301 | 2011–09–20 | PLK1 | LNP | Liver Cancer | Phase 1 Completed | National Cancer Institute | NCT01437007 |
| CALAA-01 | 2008–06–03 | R2 | Cationic lipoplex | Solid Tumors | Phase 1 Terminated | Calando Pharmaceuticals | NCT00689065 |
| DCR-MYC | 2014–04–10 | MYC | LNP | Solid Tumors, Multiple Myeloma, Lymphoma | Phase 1 Terminated | Dicerna Pharmaceuticals | NCT02110563 |
| NBF-006 | 2019–01–28 | KRAS | LNP | NSCLC, Pancreatic, CRC | Phase 1 Recruiting | Nitto BioPharma | NCT03819387 |