Table 1.
Oligonucleotide Therapeutics in the Clinic Discussed in This Review
| therapeutic | class/type | target | disease | clinical trial status | refs |
|---|---|---|---|---|---|
| Vitravene | antisense | cytomegalovirus mRNA | AIDS patients with CMV | approved | 76–78 |
| Mipomersen | antisense | Apolipoprotein B mRNA | homozygous familial hypercholesterolemia | approved | 79–87, 91, 92 |
| Eteplirsen | antisense | dystrophin Splice site | Duchenne muscular dystrophy | provisional approval | 99–103 |
| Nusinersen | antisense | SMN2 intron 7 | spinal muscular atrophy | approved | 110–115 |
| NCT02341560 | siRNA | caspase-2 | nonarteritic ischemic optic neuropathy (NAION) | III | 134, 135 |
| Patisiran | siRNA | transthyretin | familial ammyloidotic polyneuropathy due to transthyretin amyloidosis (ATTR) | III | 139, 140 |
| FANG | siRNA | furin | ovarian cancer | III | 141 |
| TKM-PLK1 | siRNA | polo-like kinase | multiple cancers | II | 142 |
| siG12D LODER | siRNA | mutant Kirsten ras oncogene | pancreatic cancer | III | 143 |
| TD101 | siRNA | mutant keratin K6a | pachyonychia congentia | I | 144 |
| RXI-109 | siRNA | connective tissue growth factor | scar healing | II | 145 |
| CEQ508 | siRNA | β-Catenin | familial adenomatous polyposis | I | 146 |
| Miravirsen | antagomiR | miR-122 | hepatitis C (HCV) | II | 169, 170 |
| RG-101 | antagomiR | miR-122 | hepatitis C (HCV) | Ib | 171 |
| MRG-106 | antagomiR | miR-155 | cutaneous T-cell lymphoma | I | 175 |
| RG-125 | antagomiR | miR-103/107 | nonalcoholic steatohepatitis | 177 | |
| MesomiR-1 | miRNA mimic | miR-16 | malignant pleural mesothelioma and nonsmall-cell lung cancer | I | 190, 191 |
| MRX34 | miRNA mimic | miR-34 | nonsmall-cel lung cancer | halted | 195–197 |
| Pegaptanib | aptamer | vascular endothetial growth factor | wet age-related macular degeneration | approved | 223–227 |
| REG1 | aptamer | coagulation factor IX a | coronary artery disease | terminated in phase III | 228, 229, 233–239 |
| ARC1779 | aptamer | von Willebrand factor | coronary artery disease | I | 240, 242–247 |
| NOX-A12 | aptamer | stromal cell-derived factor 1 | multiple myeloma | II | 217, 251, 252 |
| NOX-E36 | aptamer | monocyte chemoattractant protein-1 | diabetic nephropathy | II | 219, 255–257 |
| OZ1 | ribozyme | HIV-1 | HIV | terminated | 276, 280–283 |
| Angiozyme | ribozyme | VEGF | metastatic breast cancer | terminated in phase II | 271, 273, 285–287 |