TABLE 1.
FDA-approved siRNA medications
| Medication | Chemistry | Target | Disease | FDA Approval | Reference |
|---|---|---|---|---|---|
| Patisiran (Onpattro) |
Sense: 5′-GUmAACmCmAAGAGUmAUmUmCmCmAUmdTdT-3′ Antisense: 3′-dTdTCAUmUGGUUCUCAUmAAGGUA-5′ |
TTR | hATTR | 2018 | (Onpattro, 2018) |
| Givosiran (Givlaari) |
Sense: 5′-CmsAmsGmAmAmAmGfAmGfUmGfUmCfUmCfAmUmCmUmUmAm-L96-3′ Antisense: 3′-UmsGmsGmUfCmUfUmUfCmUfCmAfCmAfGmAfGmUfAmGfAfsAfsUm-5′ |
ALAS1 | AHP | 2019 | (Givlaari, 2019) |
| Lumasiran (Oxlumo) |
Sense: 5′-GmsAmsCmUmUmCfAmUfCfCfUmGmGmAmAmAmUmAmUmAm-L96-3′ Antisense: 3′-AmsCmsCmUmGmAmAmAfGmUfAmGmGmAmCfCfUmUfUmAmUmsAfsUm-5′ |
HAO1 | PH1 | 2020 | (Oxlumo, 2020) |
| Inclisiran (Leqvio) |
Sense: 5′-CmsUmsAmGmAmCmCfUmGfUmdTUmUmGmCmUmUmUmUmGmUm-L96-3′ Antisense: 3′-AmsAmsGmAmUmCfUmGfGmAfCmAfAmAfAmCfGmAfAfAfAmsCfsAm-5′ |
PCSK9 | HeFH, ASCVD | 2021 | (Leqvio, 2021) |
A, adenosine; Af, adenine 2'-F ribonucleoside; Am, adenine 2'-OMe ribonucleoside; C, cytidine; Cf, cytosine 2'-F ribonucleoside; Cm, cytosine 2'-OMe ribonucleoside; dT, thymidine; G, guanosine; Gf, guanine 2'-F ribonucleoside; Gm, guanine 2'-OMe ribonucleoside; L96, tri-N-acetylgalactosamine; s, phosphorothioate; U, uracil; Uf, uracil 2'-F ribonucleoside; Um, uracil 2'-OMe ribonucleoside.