Table 1.
The siRNA/MicroRNA-Based Drugs Targeting Different Diseases in Clinical Trials
| Disease | Target | Vehicle | Drug Name | Sponsor | ClinicalTrials.gov Identifier (Phase) |
|---|---|---|---|---|---|
| Cancer | |||||
| HC, ST, ACC, GNT | siRNA target PLK1 | lipid nanoparticle | TKM 080301 | Tekmira Pharmaceuticals | NCT02191878 (I/II) |
| NCT01262235 (I/II) | |||||
| NCT01437007 (I) | |||||
| ST, MM, NHL | siRNA target MYC | lipid nanoparticle | DCR-MYC | Dicerna Pharmaceuticals | NCT02110563 (I) NCT02314052 (I/II) |
| ST | siRNA target RRM2 | polymer nanoparticle | CALAA-01 | Calando Pharmaceuticals | NCT00689065 (I) |
| ST | siRNA target EphA2 | liposome | siRNA-EphA2-DOPC | M.D.Anderson Cancer Center | NCT01591356 (I) |
| Leukemia | antisense target GRB-2 | neutral liposomes | BP1001 | Bio-Path Holdings | NCT01159028 (I) |
| ASC, PC | siRNA target PKN3 | lipid nanoparticle | Atu027 | Silence Therapeutics | NCT01808638 (I/II) NCT00938574 (I) |
| PDA, PC | siRNA target K-RAS | biodegradable polymer matrix | siG12D LODER | Silenseed | NCT01188785 (I) NCT01676259 (II) |
| Glioblastoma | siRNA target p53 | nanoparticle (NPs) | Temozolomide/SGT-53 | SynerGene Therapeutics | NCT02340156 |
| Lung cancer | siRNA target Fus1 | DOTAP-Chol | Fus1/Erlotinib | Genprex | NCT01455389 (I/II) |
| HC | siRNA target CEBPA | liposomal nanoparticle | MTL-CEBPA | Mina Alpha | NCT02716012 (I) |
| Glioblastoma | siRNA target Bcl2L12 | spherical gold nanoparticle | NU-0129 | Northwestern University | NCT03020017 (early I) |
| IMG | siRNA target UGT1A1*28 | nanoliposomal | CPT-11 | University of California, San Francisco | NCT00734682 (I) |
| Advanced, metastatic cancer, ST | shRNA STMN1 | BIV-lipoplex | pbi-shRNA STMN1 LP | Strike Bio | NCT01505153 (I) |
| Ewing’s sarcoma | shRNA EWS/FLI1 type 1 | BIV-lipoplex | pbi-shRNA EWS/FLI1 Type 1 LPX | Strike Bio | NCT02736565 (I) |
| NR | eIF5AK50R plasmid eIF5A siRNA | polyethylenimine | SNS01-T | Senesco Technologies | NCT01435720 (II) |
| MPM, NSCLC | microRNA -16 mimic target EGFR | EDV | TargomiRs | Asbestos Diseases Research Foundation | NCT02369198 (I) |
| Virus infection | |||||
| EVD | siRNA target VP24, and VP35 regions, EBOV polymerase inhibitor | lipid nanoparticle | Favipiravir | INSERM, France | NCT02329054 (II) |
| Other Disease | |||||
| Hepatic fibrosis | siRNA target HSP47 | lipid nanoparticle | ND-L02 s0201 injection | Bristol-Myers Squibb | NCT02227459 (Ib/II) |
| Hypercholesterolemia | siRNA target APOB | lipid nanoparticle | PRO-040201 | Tekmira Pharmaceuticals | NCT00927459 (I), terminated |
Source: https://clinicaltrials.gov. ACC, adrenocortical carcinoma; ASC, advanced solid cancer; GNT, gastrointestinal neuroendocrine tumors; HC, hepatocellular carcinoma; MM, multiple myeloma; MPM, malignant pleural mesothelioma; NHL, non-Hodgkins lymphoma; NSCLC, non-small-cell lung cancer; PC, pancreatic cancer; PDA, pancreatic ductal adenocarcinoma; ST, solid tumor; ALC, advanced liver cancer; SCLC, squamous cell lung cancer; IMG, intracranial malignant glioma; NR, not recorded; EGFR, epidermal growth factor receptor; GRB-2, Growth Factor Receptor Bound Protein-2; RRM2, Ribonucleotide Reductase Regulatory Subunit M2; PLK1,Polo-Like Kinase 1; HSP47, Heat Shock Protein 47; EphA2, Ephrin type-A receptor 2; eIf5A, Eukaryotic translation initiation factor 5A-1; EDV, EnGeneIC Delivery Vehicle; CEBPA,CCAAT/enhancer-binding protein alpha; BIV-lipoplex, bilamellar invaginated vesicle lipoplex; EVD, Ebola virus disease; PKN3, protein kinase N3; K-Ras oncogene, Kirsten rat sarcoma viral oncogene; APOB, apolipoprotein B; VP24, virus protein 24; VP35, virus protein 3.