Table 2.
Approved siRNA-based therapies for genetic diseases
| Trade name (Drug Name) | Approval year | Disease | Targeted genes | Vector - modification | Company |
|---|---|---|---|---|---|
| Onpattro™ (Patisiran) | 2018 | Adult patients with hereditary transthyretin mediated amyloidosis | Transthyretin (TTR) | Lipid nanoparticle | Alnylam |
| Givlaari™ (Givosiran) | 2019 | Adult patients with acute hepatic porphyria | aminolevulinate synthase 1 (ALAS1) | RNA - GalNAc-conjugation | Alnylam |
| Oxlumo™ (Lumasiran) | 2020 | Adult and pediatric patients with primary hyperoxaluria type 1 | Hydroxyacid oxidase 1 (HAO1) | RNA - GalNAc-conjugation | Alnylam |