Table 2.
Summary of current oligonucleotide therapeutics in clinical trials for oncology.
| Oligonucleotide Therapeutics | Target | Drug Modality | Cancer Types | Clinical Trials |
|---|---|---|---|---|
| DNAzyme targeting EBV-LMP1 (DZ1) | EBV-LMP1 | DNAzyme | Nasopharyngeal Cancer | NCT01449942 |
| 68Ga-Sgc8 | PTK7 | aptamer | Colorectal Cancer | NCT03385148 |
| EYE001 (Anti-VEGF Pegylated Aptamer) | VEGF | aptamer | Retinal Cancer | NCT00056199 |
| NOX-A12 | CXCL12 | aptamer | Pancreatic Cancer|Colorectal Cancer|Myeloma|Leukemia | NCT01521533|NCT01521533|NCT03168139 |
| AS1411 | NCL | aptamer | Acute Myeloid Leukemia | NCT01034410|NCT00881244|NCT00740441|NCT00512083 |
| KRAS G12D siRNA | KRASG12D | siRNA | Pancreatic Cancer | NCT03608631 |
| EphA2-targeting DOPC-encapsulated siRNA | EPHA2 | siRNA | Solid Tumors | NCT01591356 |
| APN401 | CBLB | siRNA | Brain Cancer|Melanoma|Pancreatic Cancer|Renal Cell Cancer | NCT03087591|NCT02166255 |
| Proteasome siRNA and tumor antigen RNA-transfected dendritic cells | LMP2, LMP7, MECL1 | siRNA | Melanoma | NCT00672542 |
| TKM-080301 | PLK1 | siRNA | Cancer with hepatic metastases| Liver Cancer|Hepatocellular Cancer| Adrenocortical Cancer | NCT01437007|NCT02191878|NCT01262235 |
| Atu027 | PNK3 | siRNA | Solid Tumors|Pancreatic Cancer | NCT00938574|NCT01808638 |
| DCR-MYC | MYC | siRNA | Solid Tumors|Hepatocellular Cancer | NCT02110563|NCT02314052 |
| CALAA-01 | M2 subunit of ribonucleotide reductase (R2) | siRNA | Solid Tumors | NCT00689065 |
| siG12D LODER | KRASG12D | siRNA | Pancreatic Cancer | NCT01676259|NCT01188785 |
| ARO-HIF2 | HIF2A | siRNA | Clear Cell Renal Cell Carcinoma | NCT04169711 |
| SV40 vectors carrying siRNA | Unknown | siRNA | Chronic Myeloid Leukemia | NCT00257647 |
| MRX34 | 30 unique oncogenes, including but not limited to MET, MYC, PDGFRA, CDK4/6 and BCL2 | miRNA | Liver Cancer|Lung Cancer |Lymphoma |Melanoma|Multiple Myeloma|Renal Cell Cancer| | NCT01829971|NCT02862145 |
| INT-1B3 | JNK1 | miRNA | Solid Tumor | NCT04675996 |
| TargomiRs | Multiple oncogenes, including BCL2, MCL1, CCND1, and WNT3A | miRNA | Malignant Pleural Mesothelioma|Non-Small Cell Lung Cancer | NCT02369198 |
| Cobomarsen (MRG-106) | MIR155 | miRNA | Cutaneous T-Cell Lymphoma|Lymphoma|Leukemia | NCT03837457|NCT03713320|NCT02580552 |
| 1018 ISS | TLR9 | ASO | Non-Hodgkin’s Lymphoma|Colorectal Cancer | NCT00251394|NCT00403052 |
| AEG35156 | XIAP | ASO | Hepatocellular Cancer|Pancreatic Cancer|Breast Cancer|Non-Small Cell Lung Cancer| Leukemia|Lymphoma | NCT00357747|NCT00363974|NCT00372736|NCT00385775|NCT00557596|NCT00558545|NCT00558922|NCT00768339|NCT00882869|NCT01018069 |
| Apatorsen (OGX-427) | HSP27 | ASO | Urologic Cancer|Bladder Cancer|Prostate Cancer|Urothelial Cancer|Non-Small Cell Lung Cancer | NCT00487786|NCT01454089|NCT01681433|NCT01780545|NCT01829113 |
| ARRx (AZD5312) | AR | ASO | Prostate Cancer | NCT02144051|NCT03300505 |
| AZD4785 | KRAS | ASO | Non-Small Cell Lung Cancer | NCT03101839 |
| AZD8701 | FOXP3 | ASO | Advanced Cancer | NCT04504669 |
| AZD9150 | STAT3 | ASO | Bladder Cancer|Lymphoma|Malignancies | NCT02546661|NCT02549651|NCT03394144|NCT03527147|NCT03819465 |
| BP1001 | GRB2 | ASO | Ph1 Positive Leukemia|Acute Myeloid Leukemia|Chronic Myelogenous Leukemia | NCT01159028|NCT02781883|NCT02923986|NCT04196257 |
| Cenersen (EL625) | TP53 | ASO | Acute Myelogenous Leukemia|Lymphoma | NCT00074737|NCT00636155|NCT00967512|NCT02243124 |
| CpG 7909 (PF03512676) | TLR9 | ASO | Melanoma|Breast Cancer|Renal Cancer|Lymphoma|Non-Small Cell Lung Cancer|Esophageal Cancer|Prostate Cancer | NCT00031278|NCT00040950|NCT00043368|NCT00043394|NCT00043407|NCT00043420|NCT00070629|NCT00070642|NCT00085189|NCT00112242|NCT00145145|NCT00185965|NCT00199836|NCT00226993|NCT00233506|NCT00292045|NCT00299728|NCT00369291|NCT00438880|NCT00471471|NCT00490529|NCT00669292|NCT00819806|NCT00824733|NCT00880581|NCT01266603|NCT01588015 |
| CpG ODN (GNKG168) | TLR9 | ASO | Leukemia | NCT01035216|NCT01743807 |
| CpG Oligonucleotide 1 | TLR9 | ASO | Breast Cancer | NCT00640861 |
| CpG-ODN 1 | TLR9 | ASO | Glioblastoma | NCT00190424 |
| Custirsen (OGX-011) | ApoJ | ASO | Prostate Cancer|Breast Cancer|Non-Small Cell Lung Cancer | NCT00054106|NCT00138658|NCT00138918|NCT00258375|NCT00258388|NCT00327340|NCT00471432|NCT01083615|NCT01188187|NCT01497470|NCT01578655|NCT01630733 |
| Danvatirsen (AZD9150, ISIS STAT3Rx) | STAT3 | ASO | Advanced Cancers | NCT01563302|NCT01839604|NCT02417753|NCT02417753|NCT02499328|NCT02983578|NCT03334617 |
| EGFR Antisense DNA | EGFR | ASO | Head and Neck Squamous Cell Cancer|Gastric Cancer|Ovarian Cancer|Prostate Cancer | NCT00009841|NCT00023634|NCT00903461|NCT01592721|NCT03433027 |
| EZN-2968 (RO7070179,SPC2968) | HIF1A | ASO | Hepatocellular Cancer|Lymphoma | NCT00466583|NCT01120288|NCT02564614|NCT00466583 |
| G4460 | CMYB | ASO | Leukemia| Hematologic Malignancies | NCT00002592| NCT00780052 |
| IGF-1R/AS ODN | IGF1 | ASO | Glioma | NCT01550523|NCT02507583 |
| IGV-001 containing autologous GBM cells treated with antisense oligonucleotide (IMV-001) | IGF1R | ASO | Glioblastoma | NCT04485949 |
| IMO-2055 (EMD 1201081) | TLR9 | ASO | Renal Cell Cancer|Colorectal Cancer|Non-Small Cell Lung Cancer|Head and Neck Cancer | NCT00729053|NCT01040832|NCT00633529|NCT00719199|NCT01360827 |
| ION251 | IRF4 | ASO | Myeloma | NCT04398485 |
| ION537 | YAP1 | ASO | Advanced Solid Tumors | NCT04659096 |
| ISIS 183750(ISIS-EIF4ERx, LY2275796) | EIF4E | ASO | Castrate-Resistant Prostate Cancer|Non-Small Cell Lung Cancer|Colorectal Cancer | NCT00903708|NCT01234025|NCT01234038|NCT01675128 |
| ISIS 2503 | HRAS | ASO | Colorectal Cancer|Pancreatic Cancer | NCT00004193|NCT00005594|NCT00006467 |
| ISIS 5132 | CRAF | ASO | Ovarian Cancer | NCT00003892 |
| L-Bcl-2 antisense oligonucleotide | BCL2 | ASO | Advanced Lymphoid Malignancies | NCT04072458 |
| LErafAON | CRAF | ASO | Cancers | NCT00024648|NCT00024661|NCT00100672 |
| Lucanix | TGFB2 | ASO | Non-small Cell Lung Cancer | NCT01058785|NCT01279798 |
| LY2181308 | BIRC5 | ASO | Non-small Cell Lung Cancer | NCT01107444 |
| LY900003 (ISIS 3521, Affinitak) | PKCA | ASO | Melanoma|Lung Cancer|Non-Small Cell Lung Cancer|Breast Cancer | NCT00003989|NCT00017407|NCT00034268|NCT00042679|NCT00042679|NCT00003236 |
| MTL-CEBPA | CEBPA | ASO | Hepatocellular Cancer | NCT02716012|NCT04105335|NCT04710641 |
| Oblimersen (G3139) | BCL2 | ASO | Cancers | NCT00003103|NCT00004862|NCT00004870|NCT00005032|NCT00016263|NCT00017251|NCT00017589|NCT00017602|NCT00021749|NCT00024440|NCT00030641|NCT00039117|NCT00039481|NCT00042978|NCT00047229|NCT00049192|NCT00049374|NCT00054548|NCT00054639|NCT00055822|NCT00059813|NCT00060112|NCT00062244|NCT00063934|NCT00064259|NCT00070083|NCT00070343|NCT00078234|NCT00079131|NCT00080847|NCT00085124|NCT00085228|NCT00086944|NCT00091078|NCT00301795|NCT00409383|NCT00517218|NCT00518895|NCT00542893|NCT00543205|NCT00543231|NCT00636545|NCT00736450|NCT01200342 |
| OGX-427 | HSP27 | ASO | Cancers | NCT00487786|NCT00959868|NCT01120470|NCT01844817|NCT02423590 |
| PNT2258 | BCL2 | ASO | Prostate Cancer|Lymphoma|Melanoma | NCT01191775|NCT01733238|NCT02226965 |
| SPC2996 | BCL2 | ASO | Chronic Lymphocytic Leukemia | NCT00285103 |
| TGFβ2 Antisense-GMCSF Gene Modified Autologous Tumor Cell (TAG) Vaccine | TGFB2 | ASO | Advanced Cancer | NCT00684294 |
| SD-101 | TLR9 | ASO | Cancers | NCT01042379|NCT01745354|NCT02254772|NCT02266147|NCT02521870|NCT02731742|NCT02927964|NCT03007732|NCT03322384|NCT03410901|NCT04050085|NCT03831295 |
| Trabedersen (AP 12009, OT-101) | TGFB2 | ASO | Glioblastoma|Anaplastic Astrocytoma|Pancreatic Cancer|Melanoma|Colorectal Cancer | NCT00431561|NCT00761280|NCT00844064 |
| VEGF-Antisense Oligonucleotide | VEGF | ASO | Mesothelioma | NCT00668499 |
1 as no additional information is available about these two CpG oligonucleotides on ClinicalTrials.gov accessed on 15 March 2021, we treat them as two different CpG oligonucleotides.