Table 4.
Current status of nonviral vector-based RNA therapies in clinical trials.
| Delivery system | Intervention | RNA | Target gene | Delivery route | Sponsor | Indications | Development phase | ClinicalTrials.gov Identifier |
|---|---|---|---|---|---|---|---|---|
| Naked siRNA | ALN-RSV01 | siRNA | Nucleocapsid gene of RSV | Nebulization | Alnylam Pharmaceuticals | RSV infections | II | NCT00658086 |
| Lipid nanoparticles | TKM-080301 | siRNA | Polo-like kinase 1 (PLK1) | Hepatic intra-arterial | National Cancer Institute | Primary or secondary liver cancer | I | NCT01437007 |
| Lipid nanoparticles | TKM-080301 | siRNA | PLK1 | Intravenous | Arbutus Biopharma Corporation | Neuroendocrine tumors, adrenocortical carcinoma | I/II | NCT01262235 |
| Lipid nanoparticles | TKM-080301 | siRNA | PLK1 | Intravenous | Arbutus Biopharma Corporation | Advanced hepatocellular carcinoma | I/II | NCT02191878 |
| DOPC liposomes | siRNA-EphA2-DOPC | siRNA-EphA2 | Ephrin type-A receptor 2 (EphA2) | Intravenous | M.D. Anderson Cancer Center | Advanced Cancers | I | NCT01591356 |
| Lipid-based formulation (Cationic lipid AtuFECT, fusogenic lipid, PEG-LIPID) | Atu027 + gemcitabine | siRNA (Atu027) | Protein kinase N3 (PKN3) | Intravenous | Silence Therapeutics GmbH | Advanced or metastatic pancreatic cancer | I/II | NCT01808638 |
| PEI | RGT100-PEI | RGT100 | RIG-I | Intratumorally/intralesionally | Rigontec GMBH | Advanced Solid Tumors | I/II | NCT03065023 |
| Vitamin A-coupled lipid nanoparticle | ND-L02-s0201 | siRNA | Serpin peptidase inhibitor clade H member 1 (SERPINH1) (Hsp47) | Intravenous | Bristol-Myers Squibb | Moderate to extensive hepatic fibrosis | I | NCT02227459 |
| EnCore lipid nanoparticles | DCR-MYC | siRNA | MYC | Intravenous | Dicerna Pharmaceuticals, Inc. | Solid tumors, multiple myeloma, or Non-Hodgkins lymphoma | I | NCT02110563 |
| EnCore lipid nanoparticles | DCR-MYC | siRNA | MYC | Intravenous | Dicerna Pharmaceuticals, Inc. | Hepatocellular carcinoma | I/II | NCT02314052 |
| PLGA matrix | siG12D LODER | siRNA | G12D-mutated KRAS | Intratumoral implant | Silenseed Ltd | Unresectable locally advanced pancreatic cancer | I | NCT01188785 |
| PLGA matrix | siG12D-LODER | siRNA | G12D-mutated KRAS | Intratumoral implant | Silenseed Ltd | Unresectable locally advanced pancreatic cancer | II | NCT01676259 |