Table 2.
RNA drugs in clinical trials.
| Drug | Target | Vector | Conditions | Stage | Date | NCT Number |
|---|---|---|---|---|---|---|
| siRNA | ||||||
| Mesenchymal Stromal Cells-derived Exosomes with KRAS G12D siRNA | KrasG12D | Exosome | KRAS NP_004976.2:p.G12D Metastatic Pancreatic Adenocarcinoma Pancreatic Ductal Adenocarcinoma Stage IV Pancreatic Cancer AJCC v8 |
Phase 1 | January 27, 2021-March 31, 2022 | NCT03608631 |
| BMS-986263 | HSP47 | LNP | NASH | Phase 2 | March 17, 2021-January 9, 2024 | NCT04267393 |
| NBF-006 | GSTP | LNP | NSCLC; Colorectal Cancer; | Phase 1 | March 18, 2019-March 2023 | NCT03819387 |
| siG12D-LODER | KRAS | LODER Polymer | Pancreatic Ductal Adenocarcinoma; Pancreatic Cancer |
Phase 2 | March 7, 2018-August 2023 | NCT01676259 |
| STP705 | TGF-β1 COX-2 |
Histidine-lysine copolymer PNPs | Keloid | Phase 2 | April 29, 2021-April 29, 2021 | NCT04844840 |
| ALN-AGT01 | AGT | GalNAc conjugated | Hypertension | Phase 2 | June 25, 2021-December 2024 | NCT04936035 |
| ALN-VSP02 | KSP VEGF |
SNALP | Solid Tumors | Phase 1 | July 2010–September 2012 | NCT01158079 |
| Atu027 | PKN3 | Cationic lipoplex | Carcinoma, Pancreatic Ductal | Phase 1/2 | March 2013–January 2016 | NCT01808638 |
| mRNA | ||||||
| BI 1361849 | NY-ESO-1 MAGE-C2 MAGE-C1 Survivin 5 T4 MUC1 |
Cationic protein protamine | Metastatic NSCLC; NSCLC | Phase 1/2 | December 20, 2017-December 2024 | NCT03164772 |
| BNT162b2 | Membrane-anchored prefusion-stabilized spike protein of SARS-CoV-2 | LNP | SARS-CoV-2 Infection, COVID-19 | Phase 2/3 | March 24, 2021-September 27, 2023 | NCT04816643 |
| mRNA-2416 | OX40L | LNP | Relapsed/Refractory Solid Tumor Malignancies or Lymphoma; Ovarian Cancer | Phase 1/2 | August 9, 2017-September 20, 2022 | NCT03323398 |
| BNT113 | HPV16+ | Liposome | Unresectable Head and Neck Squamous Cell Carcinoma; Metastatic Head and Neck Cancer; Recurrent Head and Neck Cancer | Phase 2 | January 7, 2021-May 2025 | NCT04534205 |
| BNT112 | RBL038 RBL039 RBL-040 RBL-041 RBL-045 |
LPX | Prostate Cancer | Phase 1/2 | December 19, 2019-July 2023 | NCT04382898 |
| pp65-shLAMP DC with GM-CSF | pp65 | DCs | Glioblastoma Multiforme; Glioblastoma; Malignant Glioma; Astrocytoma, Grade IV; GBM | Phase 2 | August 2016–June 2024 | NCT03291002 |
| CV7202 | RABV-G | LNP | Rabies | Phase 1 | October 12, 2018-January 2023 | NCT03713086 |
| SARS-CoV-2 mRNA Vaccine | RBD of the SARS-CoV-2 spike protein | LNP | COVID-19; SARS-CoV-2 | Phase 3 | May 28, 2021-May 30, 2023 | NCT04847102 |
| mRNA-4157 | 34 neoantigens | LNP | Melanoma | Phase 2 | July 18, 2019-June 30, 2024 | NCT03897881 |
| miRNA | ||||||
| Remlarsen (MRG-201) | miR-29 | cholesterol-conjugated | Keloid | Phase 2 | June 11, 2018-June 24, 2020 | NCT03601052 |
| TargomiRs | miR-16 mimic | EDVs | Malignant Pleural Mesothelioma; NSCLC | Phase 1 | September 2014–January 4, 2017 | NCT02369198 |
| MRX34 | miR-34 | LNP | Primary Liver Cancer; SCLC; Lymphoma; Melanoma; Multiple Myeloma; Renal Cell Carcinoma; NSCLC | Phase 1 | April 2013–May 2017 | NCT02862145 |
| allogenic mesenchymal stem cells derived exosome enriched by miR-124 | miR-124 | Exosome | Cerebrovascular Disorders | Phase 1/2 | April 17, 2019-December 17, 2021 | NCT03384433 |
| INT-1B3 | miR-193a-3p | LNP | Solid Tumor | Phase 1 | December 18, 2020-December 2024 | NCT04675996 |
| ASO | ||||||
| Pelacarsen (TQJ230) |
Apo(a) | GalNAc conjugated | Cardiovascular Disease and Lipoprotein(a) | Phase 3 | December 12, 2019-June 27, 2024 | NCT04023552 |
| AKCEA-APOCIII-LRx | ApoC-III | GalNAc conjugated | Familial Chylomicronemia Syndrome | Phase 3 | November 18, 2020-June 2023 | NCT04568434 |
| IONIS-FB-LRx | Factor B | GalNAc conjugated | Primary IgA Nephropathy | Phase 2 | December 4, 2019-December 2023 | NCT04014335 |
| CiVi007 | PCSK9 | Locked nucleic acid (LNA) | Hypercholesterolemia | Phase 1 | February 7, 2018-August 18, 2020 | NCT03427710 |
| BP1001-A (Liposomal Grb2 Antisense Oligonucleotide) | GRB2 | Liposome | Solid Tumor, Adult; Carcinoma, Ovarian Epithelial; Fallopian Tube Neoplasms; Endometrial Cancer; Peritoneal Cancer; Solid Tumor | Phase 1 | August 2021–October 2022 | NCT04196257 |
| TGF-β2 antisense oligonucleotide | TGF-β2 | LNA | Primary Open Angle Glaucoma | Phase 1 | April 2015–August 2017 | NCT02406833 |
| Alicaforsen | ICAM1 | 2′-H modified | Pouchitis | Phase 3 | December 3, 2015-October 29, 2018 | NCT02525523 |
| OGX-427 | HSP27 | 2′-MOE modified | Urologic Neoplasms; Metastatic Bladder Cancer; Urinary Tract Neoplasms | Phase 2 | October 2011–November 2014 | NCT01454089 |
| Miravirsen | miR-122 | LNA | Hepatitis C | Phase 2 | November 2012–April 2014 | NCT01727934 |
All the information from https://clinicaltrials.gov/.
Abbreviation: HSP47: heat shock proteins 47, NASH: Nonalcoholic Steatohepatitis, GSTP: Glutathione S-transferase Pi, NSCLC: non-small cell lung cancer, LODER: Local Drug EluteR, TGF-β: transforming growth factor-beta, COX-2: cyclooxygenase-2, PNPs: polypeptide Nanoparticles, AGT: Angiotensinogen, KSP: kinesin spindle protein, PKN3: protein kinase N3, NY-ESO-1: cancer/testis antigen 1B, MAGE-C2: melanoma-associated antigen family C2, 5 T4: oncofetal antigen, MUC1: mucin 1, OX40L: OX40 ligand, HPV: Human papillomavirus, RABV-G: rabies virus glycoprotein, SCLC: small cell lung cancer, EDVs: EnGeneIC Dream Vectors (nonliving bacterial minicells), PCSK9: Proprotein convertase subtilisin/Kexin type 9, GRB-2: growth factor receptor-bound protein-2, ICAM1: intercellular adhesion molecule 1.