Table 3.
Nucleic acid-based clinical trials in progress for anticancer therapy
| Drugs | Molecular target(s) | Cancer type(s) | Stand-alone/in combination | Delivery system | Route(s) of administration | Phase | Clinical trial identifier(s) |
|---|---|---|---|---|---|---|---|
| Immunomodulators | |||||||
| SB11285 | STING | advanced solid tumors | atezolizumab | naked | IV | 1 | NCT04096638 |
| CRD3874-SI | STING | advanced solid tumors | single agent | naked | IV | 1 | NCT06021626 |
| Dazostinag | STING | advanced solid tumors | single agent/with pembrolizumab | naked | IV | 1 | NCT04420884, NCT04879849 |
| TAK-500 | STING | advanced solid tumors | single agent/with pembrolizumab | anti-CCR2 antibody conjugation | IV | 1 | NCT05070247 |
| KL340399 | STING | advanced solid tumors | single agent | naked | ITU | 1 | NCT05549804 |
| DEC-C | RIG-I | melanoma | nivolumab | naked | oral | 1b/2 | NCT05089370 |
| Poly-IC12U | TLR-3 | pancreatic cancer/melanoma/TNBC/ovarian cancer | single agent/with celecoxib, various immunotherapies | naked | IV/IP | 1/2 | NCT05494697, NCT04093323, NCT05927142, NCT05756166, NCT02432378 |
| BDC-1001 | TLR-7, -8 | advanced HER2-expressing solid tumors | single agent/with nivolumab | anti-HER2 antibody conjugation | not provided | 1 | NCT04278144 |
| BDB018 | TLR-7, -8 | advanced solid tumors | single agent/with pembrolizumab | naked | IV | 1 | NCT04840394 |
| EIK1001 | TLR-7, -8 | NSCLC | single agent/with pembrolizumab, various chemotherapies | naked | not provided | 2 | NCT06246110 |
| SD-101 | TLR-9 | metastatic uveal melanoma in the liver/pancreatic cancer | single agent/with various immunotherapies | naked | hepatic artery infusion/pancreatic retrograde venous infusion | 1/1b | NCT04935229, NCT05607953 |
| CpG-ODN | TLR-9 | advanced solid tumors | CAR-T cells secreting scFv against OX40 | naked | ITU | 1 | NCT04952272 |
| Aptamers | |||||||
| AS1411 | nucleolin | metastatic renal cell carcinoma | single agent | naked | IV | 2 | NCT00740441 |
| AM003 | personalized | advanced solid tumors | single agent | naked | ITU | 1 | NCT06258330 |
| NOX-A12 | CXCL12 | glioblastoma/metastatic pancreatic cancer | radiotherapy, bevacizumab, pembrolizumab, various chemotherapies | naked | IV | 1/2 | NCT04121455, NCT04901741 |
| siRNAs | |||||||
| NUDT21 siRNA | NUDT21 | retinoblastoma | single agent | naked | intravitreal | 1 | NCT06424301 |
| NBF-006 | KRAS mutation | NSCLC, pancreatic cancer and colorectal cancer | single agent | naked | IV | 1 | NCT03819387 |
| EphA2 siRNA | EphA2 | advanced or recurrent solid tumors | single agent | DOPC | IV | 1 | NCT01591356 |
| Cbl-b siRNA | Cbl-b | advanced solid tumors | single agent | PBMCs | IV | 1b | NCT06172894 |
| siG12D LODER | KRAS G12D | pancreatic cancer | various chemotherapies | polymeric matrix | ITU | 2 | NCT01676259 |
| iExosomes | KRAS G12D | metastatic pancreatic cancer | single agent | MSC exosomes | IV | 1 | NCT03608631 |
| ASOs | |||||||
| Danvatirsen | STAT3 | pancreatic cancer, NSCLC, and colorectal cancer | single agent/with durvalumab | naked | IV | 2 | NCT02983578 |
| BP1001-A | Grb2 | advanced or recurrent solid tumors | single agent/with paclitaxel | liposomes | IV | 1 | NCT04196257 |
| BP1001 | Grb2 | acute myeloid leukemia | ventoclax and decitabine | liposomes | IV | 2 | NCT02781883 |
| WGI-0301 | Akt-1 | advanced solid tumors | single agent | LNP | not provided | 1 | NCT05267899 |
| AZD8701 | FOXP3 | advanced solid tumors | single agent/with durvalumab | naked | IV | 1 | NCT04504669 |
| IMV-001 | IGF-1R | glioblastoma | radiation therapy and temozolomide | personalized whole-tumor-derived cells | implantation with biodiffusion chambers | 2b | NCT04485949 |
| BP1002 | BCL-2 | advanced lymphoid malignancies/acute myeloid leukemia | single agent/with decitabine | liposomes | IV | 1/1b | NCT04072458, NCT05190471 |
| OT-101 | TGF-β2 | NSCLC/pancreatic cancer | single agent/with pembrolizumab, various chemotherapies | naked | IV | 1/2/3 | NCT06579196, NCT06079346 |
| TASO-001 | TGF-β2 | advanced or metastatic solid tumors | aldesleukin | naked | IV | 1 | NCT04862767 |
BCL-2, B cell lymphoma 2; CAR-T, chimeric antigen receptor T cell; Cbl-b, casitas B lymphoma-b; CCR2, C-C motif chemokine receptor 2; DOPC, 1,2-dioleoyl-sn-glycero-3-phosphocholine; EphA2, ephrin type-A receptor 2; FOXP3, forkhead box P3; Grb2, growth factor receptor bound protein 2; IGF-1R, type 1 insulin-like growth factor receptor; IP, intraperitoneal infusion; ITU, intratumoral injection; IV, intravenous infusion; LNP, lipid nanoparticle; NSCLC, non-small cell lung cancer; NUDT21, nudix hydrolase 21; PBMCs, peripheral blood mononuclear cells; STAT3, signal transducer and activator of transcription 3; TGF-β2, transforming growth factor β; TNBC, triple-negative breast cancer.