TABLE 2.
Therapeutic siRNAs in clinical trials
| Product | Sponsor Company | Target | Disease | Current Development |
|---|---|---|---|---|
| Fitusiran (ALN-AT3SC) | Sanofi Genzyme | Antithrombin | Hemophilia A and B | Phase III – NCT03417245 (ATLAS-A/B) Phase III – NCT03417102 (ATLAS-INH) Phase III – NCT03549871 (ATLAS-PPX) Phase II/III – NCT03974113 (ATLAS-PEDS) Phase III – NCT03754790 (ATLAS-OLE) |
| Nedosiran (DCR-PHXC) | Dicerna Pharmaceuticals | Hepatic LDH | Primary hyperoxaluria | Phase III – NCT04042402 (PHYOX3) Phase II – NCT05001269 (PHYOX8) |
| Teprasiran (QPI-1002) | Quark Pharmaceuticals | p53 | AKI | Phase III – NCT02610296 (ReGIFT) |
| Tivanisiran (SYL-1001) | Sylentis S.A. | Transient receptor potential cation channel subfamily V member 1 | Ocular pain, DED | Phase III – NCT03108664 (HELIX) |
| Vutrisiran (ALN-TTRSC02) | Alnylam Pharmaceuticals | Transthyretin | hATTR | Phase III – NCT03759379 (HELIOS-A) Phase III – NCT04153149 (HELIOS-B) |
| Cosdosiran (QPI-1002) | Quark Pharmaceuticals | Caspase-2 | NAION | Phase II/III (terminated) – NCT02341560 |
| AGN211745(Sirna-027) | Allergan | VEGFR1 | CNV, AMD | Phase I/II – NCT00363714 |
| PF-04523655 | Quark Pharmaceuticals | VEGFR1 | AMD | Phases I – NCT00725686 |
| AMD | Phase II – NCT00713518 | |||
| CNV, RN, DME | Phase II – NCT01445899 | |||
| siG12D LODER | Silenseed Ltd | KRASG12D | PDAC, pancreatic cancer | Phase I – NCT01188785 Phase II – NCT01676259 |
| TKM-080301 | Arbutus Biopharma Corporation | PLK1 | HCC | Phases I/II – NCT02191878 |
| Neuroendocrine tumors, ACC | Phase I/II – NCT01262235 | |||
| Atu027 | Silence Therapeutics GmbH | Protein kinase N3 | Advanced solid tumors | Phase I – NCT00938574 |
| PDC | Phases I/II – NCT01808638 | |||
| TD101 | Pachyonychia Congenita Project | KRT6A | Pachyonychia congenita | Phase I – NCT00716014 |
| ARC-520 | Arrowhead Pharmaceuticals | cccDNA-derived viral mRNA | Safety/tolerability in healthy volunteers | Phase I – NCT01872065 |
| QPI-1002 | Quark Pharmaceuticals | p53 | ARF, AKI | Phase I – NCT00554359 |
| DGF, complications of kidney transplant | Phases I/II – NCT00802347 |
ACC, adrenocortical carcinoma; AMD, age-related macular degeneration; ARF, acute renal failure; CNV, choroidal neovascularization; DGF, delayed graft function; DME, diabetic macular edema; DR, diabetic retinopathy; HCC, hepatocellular carcinoma; LDH, lactate dehydrogenase; NAION, nonarteritic anterior ischemic optic neuropathy; PDAC, pancreatic ductal adenocarcinoma; PDC, pancreatic ductal carcinoma; VEGFR1, vascular endothelial growth factor 1.