Cemdisiran is an N-acetylgalactosamine (GalNAc) conjugated siRNA for the treatment of complement-mediated diseases by suppressing liver production ofcomplement 5 (C5)protein.

16782.53

Solid

C₅₄₂H₇₁₁F₁₇N₁₆₉O₃₃₀P₄₅S₆

1639264-46-2

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)

Room temperature in continental US; may vary elsewhere.

4°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)