Table 6.
Approval Date | Company | Therapeutic Name | Disease | Target | Delivery Route | Chemical Modification | Delivery System |
---|---|---|---|---|---|---|---|
August 2018 | Alnylam | Patisiran | hATTR | TTR | Intravenous | 2′-OMe modification | Second-generation LNPs |
November 2019 | Alnylam | Givosiran | AHP | ALAS1 | Subcutaneous | PS linkages, 2′-OMe, 2′-F modification |
GalNAc ligand conjugate |
November 2020 | Alnylam | Lumasiran | PH1 | HAO1 | Subcutaneous | PS linkages, 2′-OMe, 2′-F modification |
GalNAc ligand conjugate |
December 2020 | Alnylam Novartis |
Inclisiran | ACD | PCSK9 | Subcutaneous | PS linkages, 2′-OMe, 2′-OMOE, 2′-F modification | GalNAc ligand conjugate |
Note: Second-generation LNPs, containing CHOL, a polar lipid 1,2-distearoyl-sn-glycero-3-phosphocholine, PEGlated lipid PEG2000-C-DMG, ionizable amino lipid Dlin-MC3-DMA; AHP, acute hepatic porphyria; GalNAc, N-acetylgalactosamine.