Table 1.
The working mechanisms and target diseases of approved RNA drugs described in this paper.
| RNA drug | Brand name | Approved year | Action mechanism | Target disease |
|---|---|---|---|---|
| Antisense oligonucleotides | ||||
| Fomivirsen | Vitravene | 1998 | Inhibition of the translation of viral mRNA encoding IE2 protein | CMV retinitis |
| Mipomersen | Kynamro | 2013 | Induction of the degradation of APOB mRNA | Familial hypercholesterolemia |
| Nusinersen | Spinraza | 2016 | Induction of exon inclusion in SMN2 mRNA | Spinal muscular atrophy |
| Eteplirsen | Exondys 51 | 2016 | Induction of exon skipping in DMD mRNA | Duchenne muscular dystrophy |
| Inotersen | Tegsedi | 2018 | Induction of the degradation of TTR mRNA | Hereditary transthyretin amyloidosis |
| Golodirsen | Vyondys 53 | 2019 | Induction of exon skipping in DMD mRNA | Duchenne muscular dystrophy |
| Small interfering RNAs | ||||
| Patisiran | Onpattro | 2018 | RNA interference-mediated cleavage of TTR mRNA | Hereditary transthyretin amyloidosis |
| Givosiran | Givlaari | 2019 | RNA interference-mediated cleavage of ALAS1 mRNA | Acute hepatic porphyria |
| Lumasiran | Oxlumo | 2020 | RNA interference-mediated cleavage of HAO1 mRNA | Primary hyperoxaluria type 1 |
| Inclisiran | Leqvio | 2021 | RNA interference-mediated cleavage of PCSK9 mRNA | Hypercholesterolemia |
| RNA aptamers | ||||
| Pegaptanib | Macugen | 2004 | Antagonistic binding to VEGF protein | Age-related macular degeneration |
| Messenger RNAs | ||||
| Tozinameran | Comirnaty | 2020 | Induction of immune response by producing the spike protein of SARS-CoV-2 | COVID-19 |
| Elasomeran | Spikevax | 2020 | Induction of immune response by producing the spike protein of SARS-CoV-2 | COVID-19 |