Table 2.
FDA-approved therapeutic RNA.
| RNA type | Name | Target | Disease type | Company | Approved year |
|---|---|---|---|---|---|
| ASO | Nusinersen | Survival of motor neuron gene | Spinal muscular atrophy | Ionis | 2016 |
| Eteplirsen | Exon 51 of dystrophin pre-mRNA | Duchenne muscular dystrophy | Sarepta | 2016 | |
| Inotersen | 3′ untranslated region of TTR mRNA | Hereditary transthyretin-mediated amyloidosis | Ionis | 2018 | |
| Volanesoren | mRNA for apo-CIII | Familial chylomicronemia syndrome | Ionis | 2019 | |
| Golodirsen | Exon 53 of dystrophin pre-mRNA | Duchenne muscular dystrophy | Sarepta | 2019 | |
| siRNA | Parisiran | Transthyretin | Polyneuropathy caused by hATTR | Alnylam | 2018 |
| Givosiran | ALAS1 | Acute hepatic porphyria | Alnylam | 2019 | |
| Lumasiran | HAO1 mRNA | Primary hyperoxaluria type 1 | Alnylam | 2020 | |
| mRNA | BNT162b2 | Spike protein of SARS-CoV-2 | COVID-19 | BioNTech & Pfizer | 2020 |
| mRNA-1273 | Spike protein of SARS-CoV-2 | COVID-19 | Moderna | 2020 | |
| RNA aptamer | Pegatanib | VEGF-165 | Neovascular age-related macular degeneration | Bausch & Lomb | 2014 |