TABLE 1.
RNA therapeutics approved for clinical use or undergoing clinical trials.
| Drug | Type of RNA | Company | Route of delivery | Condition/Disease | Status |
| Nusinersen (Spinraza) | ASO | Ionis | Intrathecal | Spinal muscular atrophy | FDA approval in 2016 |
| Eteplirsen (Exondys 51) | ASO | Sarepta | Intravenous | Duchenne muscular dystrophy | FDA approval in 2016 |
| Inotersen (Tegsedi) | ASO | Ionis | Subcutaneous | Familial amyloid polyneuropathy | FDA approval in 2018 |
| Volanesorsen (Waylivra) | ASO | Ionis | Subcutaneous | Familial chylomicronemia syndrome | EU approval in 2019 |
| Patisiran (Onpattro) | siRNA | Alnylam | Intravenous | Polyneuropathy | FDA approval in 2018 |
| Givosiran (Givlaari) | siRNA | Alnylam | Subcutaneous | Acute hepatic porphyria | FDA approval in 2019 |
| Cobomarsen (MRG-106) | miRNA | miRagen (Viridian) | Intravenous/subcutaneous | Blood cancers | Phase II |
| Remlarsen (MRG-201) | miRNA | miRagen (Viridian) | Intradermal | Keloids | Phase II |
| MRG-110 | miRNA | miRagen (Viridian) | Intradermal | Tissue Repair | Phase I |
| Pegaptanib (Macugen) | Aptamer (RNA) | Bausch + Lomb | Intravitreal | Macular Degeneration | FDA approval in 2014 |
| Emapticap pegol (NOX-E36) | Aptamer (RNA) | NOXXON | Intravenous/Subcutaneous | Diabetic nephropathy, lung and pancreatic cancer | Phase II |
| Olaptesed pegol (NOX-A12) | Aptamer (RNA) | NOXXON | Intravenous | Brain cancer | Phase I/II |
| BNT162b2 | mRNA | BioNTech and Pfizer | Intramuscular | COVID-19 | FDA authorization for emergency use in 2020 |
| mRNA-1273 | mRNA | Moderna | Intramuscular | COVID-19 | FDA authorization for emergency use in 2020 |
| CVnCoV | mRNA | CureVac | Intramuscular | COVID-19 | Phase III |
| AZD8601 | mRNA | Moderna/Astrazeneca | Epicardial | Ischemic heart disease | Phase II |
| mRNA-1647 | mRNA | Moderna | Intramuscular | Cytomegalovirus infection | Phase II |
| P-BCMA-101 | mRNA | Poseida | Intravenous | Multiple myeloma | Phase II |
| mRNA-4157 | mRNA | Moderna | Intramuscular | Cancer | Phase II |
| mRNA-3704 | mRNA | Moderna | Intravenous | Methylmalonic aciduria | Phase I/II |
| MRT5005 | mRNA | Translate Bio | Inhalation | Cystic Fibrosis | Phase I/II |
| mRNA-2416 | mRNA | Moderna | Intratumoral | Solid tumors/lymphoma/advanced ovarian carcinoma | Phase I/II |
| BNT131 (SAR441000) | mRNA | BioNTech/Sanofi/Genmab | Intratumoral | Advanced melanoma | Phase I/II |
| Descartes-08 | mRNA | Cartesian | Intravenous | Generalized myasthenia gravis | Phase I/II |
| BNT122 | mRNA | BioNTech | Intravenous | Solid tumors | Phase I/II |
| mRNA-2752 | mRNA | Moderna | Intratumoral | Solid tumors | Phase I |
| MEDI1191 | mRNA | Moderna | Intratumoral | Solid tumors | Phase I |
| mRNA-1944 | mRNA | Moderna | Intravenous | Chikungunya infection | Phase I |
| CV8102 | mRNA | CureVac | Intratumoral | Solid tumors | Phase I |
| ARCT-810 | mRNA | Arcturus | Intravenously | Urea disorder | Phase I |
| CV7202 | mRNA | CureVac | Intramuscular | Rabies | Phase I |
| mRNA-1893 | mRNA | Moderna | Intramuscular | Zika | Phase I |
| CV9202 | mRNA | CureVac | Intradermal | Non-small cell lung cancer | Phase I |
| mRNA-5671 | mRNA | Moderna | Intravenous | Cancer | Phase I |
| BNT111 | mRNA | BioNTech | Intravenous | Advanced Melanoma | Phase I |
The table summarizes information available at ClinicalTrials.gov (https://clinicaltrials.gov/) as of January 26, 2021. ASO, antisense oligonucleotide; siRNA, small interfering RNA; miRNA, microRNA; RNA, ribonucleic acid; mRNA, messenger RNA.