Table 1.
siRNA therapeutics in clinical trials
| Sponsor | Disease | Target | Delivery system | Route | Status (identifier #) |
|---|---|---|---|---|---|
| Silenseed Ltd. | Pancreatic cancer; PDA | Kras oncogene G12D | Biodegradable polymeric matrix |
Intratumor injection |
Phase 1 NCT01188785 Phase 2 NCT01676259 |
| M.D. Anderson Cancer Center |
Advanced cancers | EphA2 | Neutral liposomes | IV | Phase 1 NCT01591356 |
| National Cancer Institute |
Hepatic metastases | PLK1 | Lipid nanoparticles | IV | Phase 1 NCT01437007 |
| Silence Therapeutics AG |
Pancreatic ductal carcinoma | PKN3 | Pegylated cationic liposomes |
IV | Phases 1 and 2 NCT01808638 |
| Silence Therapeutics AG |
Advanced solid tumors | PKN3 | Pegylated cationic liposomes |
IV | Phase 1 NCT00938574 |
| Duke University | Metastatic Melanoma | Immunoproteasome subunits beta type 9, 8, 10 |
Transfected dendritic cells |
Intradermal injection |
Phase 1 NCT00672542 |
| Hadassah Medical Organization |
Chronic Myeloid Leukemia | Bcl2 | Simian virus 40 | In vitro WBC from patients |
NA NCT00257647 |
| Quark Pharmaceuticals |
CNV Diabetic retinopathy DME |
RTP801 | Saline | Intravitreal injection |
Phase 2 NCT01445899 |
| Quark Pharmaceuticals |
Optic atrophy NAION |
Caspase-2 | Saline | Intravitreal injection |
Phase 1 NCT01064505 |
| Opko Health, Inc | DME Macular degeneration |
VEGF | Saline | Intravitreal injection |
Phase 2 NCT00306904 NCT00259753 |
| Opko Health, Inc | Macular degeneration | VEGF | Saline | Intravitreal injection |
Phase 2 NCT00259753 |
| Sylentis, S.A. | Ocular pain Dry eye syndrome |
Capsaicin receptor TrpV1 |
Ophthalmic drops | Topical | Phase 1 and 2 NCT01438281 NCT01776658 |
| Allergan | AMD, CNV | VEGFR1 | Saline | Intravitreal injection |
Phase 1 and 2 NCT00363714 |
| Sylentis, S.A. | Ocular hypertension Glaucoma |
β-2 adrenergic receptor | Ophthalmic drops | Topical | Phase 1 and 2 NCT01227291 NCT01739244 |
| Quark Pharmaceuticals |
Kidney injury Acute renal failure |
p53 | Saline | IV | Phase 1 NCT00554359 |
| Quark Pharmaceuticals |
Delayed graft function and complication of kidney transplant |
p53 | Saline | IV | Phase 1 and 2 NCT00802347 |
| Nitto Denko Corporation |
Liver fibroses | Hsp47 | Vitamin A-coupled lipid nanoparticles |
IV | Phase 1 NCT01858935 |
| Pachyonychia Congenita Project |
Pachyonychia congenita | Keratin K6a | Injection into a callus |
Phase 1 NCT00716014 |
Information is confirmed on March 6, 2014 from clinicaltrials.gov. Those listed as “terminated” are not included
PDA pancreatic ductal adenocarcinoma; CNV choroidal neovascularization; DME diabetic macular edema; NAION non-arteritic anterior ischemic optic neuropathy; AMD age-related macular degeneration; EphA2 ephrin type-A receptor 2; PLK1 polo-like kinase 1; PKN3 protein kinase N3; RRM2 ribonucleoside-diphosphate reductase subunit M2; Bcl-2 B cell lymphoma 2; RTP801 also known as Redd1; VEGF vascular endothelial growth factor; TrpV1 receptor potential cation channel subfamily V member 1; VEGFR1 vascular endothelial growth factor receptor 1; Hsp 47 heat shock protein 47; Tf transferrin; CDP cyclodextrin-containing polymer; IV intravenous injection; WBC white blood cells; NA not applicable