Table 3.
Nucleic acid drugs in a phase 3 trial (as of 30 June 2021).
| Drug Name | Type | Target | Indication | DDS | Company |
|---|---|---|---|---|---|
| Pelacarsen | ASO | Apolipoprotein A | Cardiovascular disease | GalNAc | Ionis, Novartis |
| Eplontersen | ASO | TTR | TTR amyloidosis | GalNAc | Ionis |
| APOCIII-LRx | ASO | ApoC3 | Familial chylomicronemia syndrome | GalNAc | Ionis |
| Tofersen | ASO | SOD1 | Amyotrophic lateral sclerosis | Naked | Ionis, Biogen |
| Tominersen | ASO | HTT | Huntington’s disease | Naked | Ionis, Roche |
| ION-363 | ASO | FUS | Amyotrophic lateral sclerosis |
Naked | Ionis |
| Nexagon | ASO | Connexin 43 | Corneal injury | Naked | Ocunexus |
| Fitusiran | siRNA | Antithrombin III | Hemophilia | GalNAc | Alnylam, Sanofi |
| Vutrisiran | siRNA | TTR | TTR amyloidosis | GalNAc | Alnylam |
| Nedosiran | siRNA | LDHA | Primary hyperoxaluria | GalNAc | Dicerna |
| Teprasiran | siRNA | P53 | Acute kidney injury | Naked | Quark |
| Cosdosiran | siRNA | Caspase 2 | Non-arteritic anterior ischemic optic neuropathy |
Naked | Quark |
| Tivanisiran | siRNA | TRPV1 | Dry eye | Naked | Sylentis |
| Sepofarsen | EON | CEP290 | Leber congenital amaurosis | Naked | ProQR |
| Zorecimeran | mRNA | SARS-CoV-2 | COVID-19 vaccine | LNP | CureVac |