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RNA, (Am-sp-Am-sp-(2′-deoxy-2′-fluoro)G-Cm-(2′-deoxy-2′-fluoro)A-Am-(2′-deoxy-2′-fluoro)G-Am-(2′-deoxy-2′-fluoro)U-(2′-deoxy-2′-fluoro)A-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)U-Um-Um-(2′-deoxy-2′-fluoro)A-Um-(2′-deoxy-2′-fluoro)A-Am-Um-Am), 3′-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]-1,12,19,25-tetraoxo-16-oxa-13,20,24-triazanonacos-1-yl]-4-hydroxy-2-pyrrolidinyl]methyl hydrogen phosphate], complex with RNA (Um-sp-(2′-deoxy-2′-fluoro)A-sp-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)A-Um-Am-(2′-deoxy-2′-fluoro)A-Am-(2′-deoxy-2′-fluoro)A-Am-Um-Am-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)G-Cm-Um-Um-sp-Um-sp-Um-dT-dT) (1:1) |
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Cemdisiran is an N-acetylgalactosamine (GalNAc) conjugated siRNA for the research of complement-mediated diseases by suppressing liver production of complement 5 (C5) protein. | [References]
[1] Prajakta Badri, et al. Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria. Clin Pharmacokinet. 2021;60(3):365-378. DOI:10.1007/s40262-020-00940-9
[2] Badri P, Jiang X, Borodovsky A, et al. Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria. Clin Pharmacokinet. 2021;60(3):365-378. |
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