Table 1 siRNA-based therapeutics in clinical trials.
From: Regulation of cellular gene expression by nanomaterials
Start–end date | Drug | Target | Route | Condition | Phase | Clinical trial ID | Sponsors |
|---|---|---|---|---|---|---|---|
2010–2012 | ALN-TTR01 | TTR | IV | TTR-mediated amyloidosis | I | NCT01148953 | Alnylam Pharma |
2012–2012 2012–2014 2013–2017 2013–2017 | Patisiran (ALN-TTR02) | TTR | IV | TTR-mediated amyloidosis | I II II III | NCT01559077 NCT01617967 NCT01961921 NCT01960348 | Alnylam Pharma |
2013–2015 2013–2015 2014–2017 | Revusiran (ALN-TTRSC) | TTR | SC | TTR-cardiac amyloidosis | I II II | NCT01814839 NCT01981837 NCT02292186 | Alnylam Pharma |
2011–2013 2015–2017 | siG12D LODER | KRAS G12D | EUS biopsy needle | Pancreatic ductal adenocarcinoma | I II | NCT01188785 NCT01676259 | Silenseed Ltd. |
2011–2012 2012–2014 | SYL1001 | TRPV1 | Ophth. | Ocular pain Dryeye syndrome | I I/II | NCT01438281 NCT01776658 | Sylentis, S.A. |
2011–2012 | TKM-080301 (TKM-PLK1) | Polo-kinase-1 | IV | Solid tumors with liver involvement | I | NCT01437007 | National Cancer Inst. |
2010–2014 2014–2016 | TKM-080301 (TKM-PLK1) | Polo-kinase-1 | IV | Neuroendocrine tumors, adrenocortical carcinoma Hepatocellular carcinoma | I/II I/II | NCT01262235 NCT02191878 | Tekmira Pharma |
2011–2012 | ALN-PCS02 | PCSK9 | IV | Elevated LDL-cholesterol (LDL-C) | I | NCT01437059 | Alnylam Pharma |
2014–2015 | ALN-PCSSC | PCSK9 | SC | Elevated LDL-cholesterol (LDL-C) | I | NCT02314442 | Alnylam Pharma |
2012 | TKM-100201 (TKM-Ebola) | ZEBOV L poly m., VP24, VP35 | IV | Ebola virus infection | I | NCT01518881 | Tekmira Pharma |
2013–2014 | ND-L02-s0201 | HSP47 | IV | Healthy | I | NCT01858935 | Nitto Denko Corp. |
2014–2016 | ND-L02-s0201 | HSP47 | IV | Moderate to extensive hepatic fibrosis | I | NCT02227459 | Nitto Denko Corp. |
2014–2015 | ALN-AT3Sc | AT | SC | Hemophilia A, hemophilia B | I | NCT02035605 | Alnylam Pharma |
2014–2016 | APN401 | E3 ubiquitin ligase Cbl-b | IV | Melanoma, pancreatic cancer, renal cell cancer | I | NCT02166255 | Wake Forest Univ. |
2014–2016 | DCR-MYC | MYC | IV | Hepatocellular carcinoma | I/II | NCT02314052 | Dicerna Pharma |
2014–2015 | DCR-MYC | MYC | IV | Solid tumors, multiple myeloma, non-Hodgkin lymphoma | I | NCT02110563 | Dicerna Pharma |
2015 | siRNA-EphA2-DOPC | EphA2 | IV | Advanced cancer | I | NCT01591356 | MD Anderson Cancer Center |