Table 2.
Gene silencing nanoparticles (NPs) undergoing clinical trials for cancer treatment (adapted from [9,85,86]).
| Name (Company) | Particle Type/Drug | Application/Indication | Clinical Trials. Gov (Phase) |
|---|---|---|---|
| TKM-080301 (Arbutus Biopharma) | Lipid particle targeting Polo-Like Kinase 1 (PLK1) for delivery of siRNA | Hepatocellular carcinoma | NCT02191878 (Ph I/II) |
| siRNA-EphA2-DOPC (M.D. Anderson Cancer Center) | siRNA liposome for EPHA2 knockdown | Solid tumors | NCT01591356 (Ph I) |
| PNT2258 (ProNAi Therapeutics) | Proprietary single-stranded DNAi (PNT100) encapsulated in lipid nanoparticles | Lymphomas |
NCT02378038 (Ph II) NCT02226965 (Ph II) NCT01733238 (Ph II) |
| BP1001 (Bio-Path Holdings) oligonucleotide | Growth Factor Receptor Bound Protein-2 (GRB-2) antisense encapsulated in neutral liposomes | Leukemia | NCT01159028 (Ph I) |
| DCR-MYC (Dicerna Pharmaceuticals) | DsiRNA lipid nanoparticle for MYC oncogene silencing | Solid tumors, multiple myeloma, lymphoma, or hepatocellular carcinoma |
NCT02110563 (Ph I) NCT02314052 (Ph I/II) |
| Atu027 (Silence Therapeutics GmbH) | AtuRNAi® liposomal formulation for Protein Kinase N3 (PKN3) knockdown in vascular endothelium | Pancreatic cancer | NCT01808638 (Ph I/II) |
| SGT-53 (SynerGene Therapeutics) | Cationic liposome with anti-transferrin receptor antibody, encapsulating Wildtype p53 sequence | Glioblastoma, solid tumors, or pancreatic cancer |
NCT02354547 (Ph I) NCT00470613 (Ph I) NCT02354547 (Ph I) NCT02340156 (Ph II) |
| SGT-94 (SynerGene Therapeutics) | RB94 plasmid DNA in a liposome with anti-transferrin receptor antibody | Solid tumors | NCT01517464 (Ph I) |
| MRX34 (Mirna Therapeutics) | Double-stranded RNA mimic of miR-34 encapsulated in liposomes | Liver cancer | NCT01829971 (Ph I) |
| TargomiRs (EnGeneIC) | Anti-EGFR bispecific antibody minicells (bacteria derived nanoparticles) with a miR-16 based microRNA payload | Mesothelioma and Non-small cell lung cancer | NCT02369198 (Ph I) |
| CALAA-01 (Calando Pharmaceuticals) | Polymer (targeted)/siRNA targeting Ribonucleotide Reductase Regulatory Subunit M2 (RRM2) | Melanoma | NCT00689065 (Ph I) |
| ALN-VSP (Alnylam Pharmaceuticals) | Lipid (non-targeted)/siRNA targeting VEGFA and KSP | Solid Tumors |
NCT00882180 (Ph I) NCT01158079 (Ph I) |
| Atu027 PKN3 (Silence Therapeutics) | Lipid (non-targeted)/siRNA targeting PKN3 | Advanced solid tumors and metastatic pancreatic adenocarcinoma |
NCT00938574 (Ph I) NCT01808638 (Ph I/II) |
| TKM-PLK1 (Tekmira Pharmaceuticals) | Lipid (non-targeted)/siRNA targeting PLK1 | solid tumors, hepatocellular carcinoma, gastrointestinal neuroendocrine tumors and adrenocortical carcinoma |
NCT01262235 (Ph I/II) NCT01437007 (Ph I) NCT02191878 (Ph I/II) |
| siG12D LODER (Silenseed) | Biodegradable polymer matrix/siRNA targeting K-RAS | Pancreatic Ductal Adenocarcinoma and Pancreatic cancer |
NCT01188785 Ph I) NCT01676259 (Ph II) |
| SNS01-T (Senesco Technologies) | Polyethylenimine (non-targeted)/eIF5AK50R plasmid eIF5A siRNA | NR | NCT01435720 (Ph II) |
NR: Not reported.