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. 2018 Sep 22;12:1–18. doi: 10.1016/j.omtm.2018.09.002

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.