siRNA drugs
siRNAs（小干扰 RNA）是短片段的外源性双链 RNA（dsRNA）（20-25 个核苷酸），其能够触发RNA干扰（RNAi）途径。siRNA进入细胞后发生解链，正义链被降解的同时反义链与各种蛋白质成分形成RNA诱导沉默复合物（RISC）。RISC中保留的反义链与靶基因mRNA特异性互补。同时，RISC具有核酸酶活性，能够切割并降解靶基因mRNA，从而抑制靶基因的表达。而siRNA和靶基因序列的不完全互补会导致 mRNA 翻译抑制。
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Product Name |
CAS No. |
Purity |
Chemical Structure |
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- HY-132591A
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RNA, (Am-sp-(2'-deoxy-2'-fluoro)C-sp-Am-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'- fluoro)A-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)AAm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)C-Um-Am-Gm-sp-Am-sp-Am), complex with RNA (Cm-sp-Umsp -Am-Gm-Am-Cm-(2'-deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)G-Um-dT-UmUm-Gm-Cm-Um-Um-Um-Um-Gm-Um) 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2- deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-βD-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] (1:1), sodium salt
RNA, (Am-sp-(2'-deoxy-2'-fluoro)C-sp-Am-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'- fluoro)A-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)AAm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)C-Um-Am-Gm-sp-Am-sp-Am), complex with RNA (Cm-sp-Umsp -Am-Gm-Am-Cm-(2'-deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)G-Um-dT-UmUm-Gm-Cm-Um-Um-Um-Um-Gm-Um) 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2- deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-βD-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] (1:1), sodium salt
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- HY-132591
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Inclisiran
英克司兰
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1639324-58-5 |
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Inclisiran (ALN-PCSsc) 是一种双链小干扰 RNA (siRNA) 分子，可抑制 PCSK9 的转录。Inclisiran 可用于高脂血症和心血管疾病 (CVD) 研究。
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RNA, (Am-sp-(2'-deoxy-2'-fluoro)C-sp-Am-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'- fluoro)A-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)AAm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)C-Um-Am-Gm-sp-Am-sp-Am), complex with RNA (Cm-sp-Umsp -Am-Gm-Am-Cm-(2'-deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)G-Um-dT-UmUm-Gm-Cm-Um-Um-Um-Um-Gm-Um) 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2- deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-βD-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] (1:1)
RNA, (Am-sp-(2'-deoxy-2'-fluoro)C-sp-Am-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'- fluoro)A-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)AAm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)C-Um-Am-Gm-sp-Am-sp-Am), complex with RNA (Cm-sp-Umsp -Am-Gm-Am-Cm-(2'-deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)G-Um-dT-UmUm-Gm-Cm-Um-Um-Um-Um-Gm-Um) 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2- deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-βD-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] (1:1)
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- HY-132609
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Patisiran sodium
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1386913-72-9 |
99.09% |
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Patisiran sodium 是一种双链小干扰 RNA，靶向 Transthyretin (TTR) 信使 RNA 内的序列。Patisiran sodium 特异性抑制突变体和野生型 TTR 的肝脏合成。Patisiran sodium 可用于研究遗传性 TTR 淀粉样蛋白症的研究。
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RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA (G-Um-A-ACm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1) sodium salt
RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA (G-Um-A-ACm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1) sodium salt
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- HY-132610
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Givosiran
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1639325-43-1 |
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Givosiran (ALN-AS1) 是一种小干扰 RNA，靶向肝脏氨基纤维素合酶 1 (ALAS1) 信使 RNA。Givosiran 下调 ALAS1 mRNA 并阻止神经毒性 δ-氨基纤维酸和卟啉的积累。Givosiran 可用于急性间歇性卟啉症的研究。
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RNA, (Cm-sp-Am-sp-Gm-Am-Am-Am-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)C-Am-Um-Cm-Um-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-oxa13,20,24-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)A-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)U-Gm-sp-Gm-sp-Um) (1:1)
RNA, (Cm-sp-Am-sp-Gm-Am-Am-Am-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)C-Am-Um-Cm-Um-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-oxa13,20,24-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)A-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)U-Gm-sp-Gm-sp-Um) (1:1)
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- HY-150150
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RNA, (UUCUCCGAACGUGUCACGUUU), complex with RNA, (UUAAGAGGCUUGCACAGUGCA) (1:1)
RNA, (UUCUCCGAACGUGUCACGUUU), complex with RNA, (UUAAGAGGCUUGCACAGUGCA) (1:1)
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- HY-164740
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RNA, ([1'-de(6-amino-9H-purin-9-yl)]dA-(5'→5')-sp-Am-Cm-Gm-Gm-Gm-Am-Cm-Am-
(2'-deoxy-2'-fluoro)G-(2'-deoxy-2'-fluoro)U-(2'-deoxy-2'-fluoro)A-Um-Um-Cm-Um-CmAm-Gm-Um-Im-Am-(3'→3')-sp-[1'-de(6-amino-9H-purin-9-yl)]dA), 3'-[O-[cis-4-
[(3S,8S)-17-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-3,8-bis[[[2-[2-[[2-
(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]ethoxy]ethyl]amino]carbonyl]-
1,6,11-trioxo-15-oxa-2,7,12-triazaheptadec-1-yl]cyclohexyl] hydrogen
phosphorothioate], complex with RNA (Um-sp-(2'-deoxy-2'-fluoro)C-sp-Am-sp-(2'-
deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)G-Am-Gm-Am-Am-Um-(2'-deoxy-2'-
fluoro)A-Cm-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)CCm-(2'-deoxy-2'-fluoro)G-sp-Um) (1:1)
RNA, ([1'-de(6-amino-9H-purin-9-yl)]dA-(5'→5')-sp-Am-Cm-Gm-Gm-Gm-Am-Cm-Am-
(2'-deoxy-2'-fluoro)G-(2'-deoxy-2'-fluoro)U-(2'-deoxy-2'-fluoro)A-Um-Um-Cm-Um-CmAm-Gm-Um-Im-Am-(3'→3')-sp-[1'-de(6-amino-9H-purin-9-yl)]dA), 3'-[O-[cis-4-
[(3S,8S)-17-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-3,8-bis[[[2-[2-[[2-
(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]ethoxy]ethyl]amino]carbonyl]-
1,6,11-trioxo-15-oxa-2,7,12-triazaheptadec-1-yl]cyclohexyl] hydrogen
phosphorothioate], complex with RNA (Um-sp-(2'-deoxy-2'-fluoro)C-sp-Am-sp-(2'-
deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)G-Am-Gm-Am-Am-Um-(2'-deoxy-2'-
fluoro)A-Cm-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)CCm-(2'-deoxy-2'-fluoro)G-sp-Um) (1:1)
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- HY-132610A
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Givosiran sodium
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1639325-44-2 |
98.71% |
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Givosiran sodium 是一种小干扰 RNA，靶向肝脏氨基纤维素合酶 1 (ALAS1) 信使 RNA。Givosiran sodium 下调 ALAS1 mRNA 并阻止神经毒性 δ-氨基纤维酸和卟啉的积累。Givosiran sodium 可用于急性间歇性卟啉症的研究。
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RNA, (Cm-sp-Am-sp-Gm-Am-Am-Am-(2′-deoxy-2′-fluoro)G-Am-(2′-deoxy-2′-fluoro)G-Um-(2′-deoxy-2′-fluoro)G-Um-(2′-deoxy-2′-fluoro)C-Um-(2′-deoxy-2′-fluoro)C-Am-Um-Cm-Um-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)A-(2′-deoxy-2′-fluoro)G-Am-(2′-deoxy-2′-fluoro)U-Gm-(2′-deoxy-2′-fluoro)A-Gm-(2′-deoxy-2′-fluoro)A-Cm-(2′-deoxy-2′-fluoro)A-Cm-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)U-Gm-sp-Gm-sp-Um), sodium salt (1:1:43)
RNA, (Cm-sp-Am-sp-Gm-Am-Am-Am-(2′-deoxy-2′-fluoro)G-Am-(2′-deoxy-2′-fluoro)G-Um-(2′-deoxy-2′-fluoro)G-Um-(2′-deoxy-2′-fluoro)C-Um-(2′-deoxy-2′-fluoro)C-Am-Um-Cm-Um-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)A-(2′-deoxy-2′-fluoro)G-Am-(2′-deoxy-2′-fluoro)U-Gm-(2′-deoxy-2′-fluoro)A-Gm-(2′-deoxy-2′-fluoro)A-Cm-(2′-deoxy-2′-fluoro)A-Cm-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)U-Gm-sp-Gm-sp-Um), sodium salt (1:1:43)
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- HY-143221
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AS-Inclisiran sodium
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AS-Inclisiran sodium 是 Inclisiran 的反义链。Inclisiran (ALN-PCSsc) 是一种双链小干扰 RNA (siRNA) 分子，可抑制 PCSK-9 的转录。Inclisiran 可用于高脂血症和心血管疾病 (CVD) 研究。
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AS-Inclisiran (sodium)
AS-Inclisiran (sodium)
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- HY-132588
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Lumasiran
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1834610-13-7 |
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Lumasiran (ALN-G01) 是一种 siRNA 化合物，通过靶向乙醇酸氧化酶 (glycolate oxidase) 减少肝脏草酸的产生。Lumasiran 通过沉默编码乙醇酸氧化酶的基因，消耗乙醇酸氧化酶，从而抑制草酸的合成，草酸是一种与原发性高草酸尿1型 (PH1) 表现直接相关的有毒代谢物。
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RNA, (Gm-sp-Am-sp-Cm-Um-Um-Um-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)U-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-Um-Gm-Gm-Am-Am-Am-Um-Am-Um-Am),3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]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'-deoxy2'-fluoro)A-sp-Um-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-Am-Gm-Gm-Am-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)A-Am-Am-Gm-Um-Cm-sp-Cm-sp-Am) (1:1)
RNA, (Gm-sp-Am-sp-Cm-Um-Um-Um-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)U-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-Um-Gm-Gm-Am-Am-Am-Um-Am-Um-Am),3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]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'-deoxy2'-fluoro)A-sp-Um-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-Am-Gm-Gm-Am-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)A-Am-Am-Gm-Um-Cm-sp-Cm-sp-Am) (1:1)
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- HY-143219
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SS-Inclisiran (sodium)
SS-Inclisiran (sodium)
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- HY-147425A
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RNA, (Am-sp-(2′-deoxy-2′-fluoro)U-sp-Am-(2′-deoxy-2′-fluoro)A-Cm-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)U-Gm-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)C-Am-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)A-Cm-sp-(2′-deoxy-2′-fluoro)C-sp-Gm), complex with RNA (Cm-Gm-Gm-Um-Am-Am-(2′-deoxy-2′-fluoro)U-(2′-deoxy-2′-fluoro)G-(2′-deoxy-2′-fluoro)G-Am-Cm-Am-Gm-Am-Gm-Um-Um-sp-Am-sp-Um) 5′-[O-[21-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-9,9-bis[12-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-7-mercapto-7-oxido-2,6,8-trioxa-7-phosphadodec-1-yl]-16-mercapto-16-oxido-7,11,15,17-tetraoxa-16-phosphaheneicos-1-yl] hydroxy phosphate] (1:1), sodium salt
RNA, (Am-sp-(2′-deoxy-2′-fluoro)U-sp-Am-(2′-deoxy-2′-fluoro)A-Cm-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)U-Gm-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)C-Am-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)A-Cm-sp-(2′-deoxy-2′-fluoro)C-sp-Gm), complex with RNA (Cm-Gm-Gm-Um-Am-Am-(2′-deoxy-2′-fluoro)U-(2′-deoxy-2′-fluoro)G-(2′-deoxy-2′-fluoro)G-Am-Cm-Am-Gm-Am-Gm-Um-Um-sp-Am-sp-Um) 5′-[O-[21-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-9,9-bis[12-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-7-mercapto-7-oxido-2,6,8-trioxa-7-phosphadodec-1-yl]-16-mercapto-16-oxido-7,11,15,17-tetraoxa-16-phosphaheneicos-1-yl] hydroxy phosphate] (1:1), sodium salt
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- HY-132589A
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Vutrisiran sodium
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Vutrisiran sodium 是一种肝脏导向的、小干扰RNA (siRNA) 。 Vutrisiran sodium 靶向转甲状腺素蛋白 (TTR) 的mRNA，可用于转甲状腺素蛋白 (TTR) 介导的淀粉样变性研究。
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RNA, (Um-sp-(2'-deoxy-2'-fluoro)C-sp-Um-Um-Gm-(2'-deoxy-2'-fluoro)G-UmUm-(2'-deoxy-2'-fluoro)A-Cm-Am-Um-Gm-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy2'-fluoro)A-Um-Cm-Cm-Cm-Am-sp-Um-sp-Cm), complex with RNA (Um-sp-Gm-sp-Gm-Gm-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'-fluoro)U-Gm-Um-Am-Am-Cm-Cm-Am-Am-GmAm) 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] (1:1), sodium salt
RNA, (Um-sp-(2'-deoxy-2'-fluoro)C-sp-Um-Um-Gm-(2'-deoxy-2'-fluoro)G-UmUm-(2'-deoxy-2'-fluoro)A-Cm-Am-Um-Gm-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy2'-fluoro)A-Um-Cm-Cm-Cm-Am-sp-Um-sp-Cm), complex with RNA (Um-sp-Gm-sp-Gm-Gm-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'-fluoro)U-Gm-Um-Am-Am-Cm-Cm-Am-Am-GmAm) 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] (1:1), sodium salt
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- HY-145720
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Cemdisiran
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1639264-46-2 |
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Cemdisiran是一种 GalNAc 偶联的 siRNA干扰，通过抑制肝脏补体5 (C5)蛋白的产生来研究补体介导的疾病。
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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)
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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- HY-132589
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Vutrisiran
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1867157-35-4 |
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Vutrisiran (ALN-TTRsc02) 是一种肝脏导向的、小干扰RNA (siRNA) 。 Vutrisiran 靶向转甲状腺素蛋白 (TTR) 的mRNA，可用于转甲状腺素蛋白 (TTR) 介导的淀粉样变性研究。
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RNA, (Um-sp-(2'-deoxy-2'-fluoro)C-sp-Um-Um-Gm-(2'-deoxy-2'-fluoro)G-UmUm-(2'-deoxy-2'-fluoro)A-Cm-Am-Um-Gm-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy2'-fluoro)A-Um-Cm-Cm-Cm-Am-sp-Um-sp-Cm), complex with RNA (Um-sp-Gm-sp-Gm-Gm-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'-fluoro)U-Gm-Um-Am-Am-Cm-Cm-Am-Am-GmAm) 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] (1:1)
RNA, (Um-sp-(2'-deoxy-2'-fluoro)C-sp-Um-Um-Gm-(2'-deoxy-2'-fluoro)G-UmUm-(2'-deoxy-2'-fluoro)A-Cm-Am-Um-Gm-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy2'-fluoro)A-Um-Cm-Cm-Cm-Am-sp-Um-sp-Cm), complex with RNA (Um-sp-Gm-sp-Gm-Gm-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'-fluoro)U-Gm-Um-Am-Am-Cm-Cm-Am-Am-GmAm) 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] (1:1)
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- HY-132606A
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Nedosiran sodium
奈多西兰钠盐
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2247026-22-6 |
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Nedosiran sodium 是一种靶向乳酸脱氢酶 (LDH) 的 RNA 干扰剂 (RNAi)。Nedosiran sodium 可用于研究并发终末期肾病 (ESRD)的原发性高草酸尿症 (PH)。
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RNA, (Am-sp-Um-(2′-deoxy-2′-fluoro)G-Um-(2′-deoxy-2′-fluoro)U-Gm-Um-(2′-deoxy-2′-fluoro)C-(2′-deoxy-2′-fluoro)C-(2′-deoxy-2′-fluoro)U-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)A-Um-(2′-deoxy-2′-fluoro)C-Um-Gm-Am-Gm-Cm-Am-Gm-Cm-Cm-[2′-O-[[2-[2-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]ethoxy]ethoxy]methyl]]G-[2′-O-[[2-[2-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]ethoxy]ethoxy]methyl]]A-[2′-O-[[2-[2-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]ethoxy]ethoxy]methyl]]A-[2′-O-[[2-[2-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]ethoxy]ethoxy]methyl]]A-Gm-Gm-Cm-Um-Gm-Cm), sodium salt, complex with RNA ([1-de-β-D-ribofuranosyl-1-[(2R,3R,4S,5R)-tetrahydro-4-hydroxy-5-[(hydroxymethoxyphosphinyl)methoxy]-3-methoxy-2-furanyl]]U-(4′→5′)-sp-(2′-deoxy-2′-fluoro)C-sp-(2′-deoxy-2′-fluoro)A-sp-(2′-deoxy-2′-fluoro)G-(2′-deoxy-2′-fluoro)A-Um-(2′-deoxy-2′-flu
RNA, (Am-sp-Um-(2′-deoxy-2′-fluoro)G-Um-(2′-deoxy-2′-fluoro)U-Gm-Um-(2′-deoxy-2′-fluoro)C-(2′-deoxy-2′-fluoro)C-(2′-deoxy-2′-fluoro)U-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)A-Um-(2′-deoxy-2′-fluoro)C-Um-Gm-Am-Gm-Cm-Am-Gm-Cm-Cm-[2′-O-[[2-[2-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]ethoxy]ethoxy]methyl]]G-[2′-O-[[2-[2-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]ethoxy]ethoxy]methyl]]A-[2′-O-[[2-[2-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]ethoxy]ethoxy]methyl]]A-[2′-O-[[2-[2-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]ethoxy]ethoxy]methyl]]A-Gm-Gm-Cm-Um-Gm-Cm), sodium salt, complex with RNA ([1-de-β-D-ribofuranosyl-1-[(2R,3R,4S,5R)-tetrahydro-4-hydroxy-5-[(hydroxymethoxyphosphinyl)methoxy]-3-methoxy-2-furanyl]]U-(4′→5′)-sp-(2′-deoxy-2′-fluoro)C-sp-(2′-deoxy-2′-fluoro)A-sp-(2′-deoxy-2′-fluoro)G-(2′-deoxy-2′-fluoro)A-Um-(2′-deoxy-2′-flu
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- HY-132613
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Lumasiran sodium
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1834612-06-4 |
99.21% |
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Lumasiran sodium 是一种研究性 RNA 干扰 (RNAi) 剂，通过靶向乙醇酸氧化酶(glycolate oxidase) 减少肝脏草酸的产生。Lumasiran sodium 减少尿草酸排泄，这是原发性高草酸尿 1 型 (PH1) 进行性肾衰竭的原因。
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RNA, (Gm-sp-Am-sp-Cm-Um-Um-Um-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'- fluoro)U-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-Um-Gm-Gm-Am-Am-Am-Um-Am-Um-Am),3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]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'-deoxy2'-fluoro)A-sp-Um-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-Am-Gm-Gm-Am-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'- fluoro)A-Am-Am-Gm-Um-Cm-sp-Cm-sp-Am) (1:1) sodium salt
RNA, (Gm-sp-Am-sp-Cm-Um-Um-Um-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'- fluoro)U-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-Um-Gm-Gm-Am-Am-Am-Um-Am-Um-Am),3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]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'-deoxy2'-fluoro)A-sp-Um-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-Am-Gm-Gm-Am-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'- fluoro)A-Am-Am-Gm-Um-Cm-sp-Cm-sp-Am) (1:1) sodium salt
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- HY-150223
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GalNAc unconjugated/naked Inclisiran
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GalNAc unconjugated/naked Inclisiran 是一种没有偶联 GalNAc 的双链小干扰 RNA (siRNA) 分子，可抑制 PCSK-9 的转录。GalNAc unconjugated/naked Inclisiran 可用于高脂血症和心血管疾病 (CVD) 研究。
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GalNAc unconjugated/naked Inclisiran
GalNAc unconjugated/naked Inclisiran
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- HY-132587
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Fitusiran
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1499251-18-1 |
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Fitusiran (ALN-AT3SC) 是一种小干扰 RNA，专门针对抗凝血酶 (AT) 信使 RNA，以降低肝脏中 AT 的产生。Fitusiran 增加凝血酶的生成，并可用于血友病的研究。
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RNA, ((2'-deoxy-2'-fluoro)G-sp-Gm-sp-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)A-Am- (2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)A-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)A),3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]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)U-sp-Gm-(2'-deoxy2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)A-Um-Gm-Gm-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)C-Cm-sp-Am-sp-Gm)
RNA, ((2'-deoxy-2'-fluoro)G-sp-Gm-sp-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)A-Am- (2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)A-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)A),3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-Dgalactopyranosyl]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)U-sp-Gm-(2'-deoxy2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)A-Um-Gm-Gm-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)C-Cm-sp-Am-sp-Gm)
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- HY-143220
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SS(no Galnac)-Inclisiran (sodium)
SS(no Galnac)-Inclisiran (sodium)
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- HY-132596A
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RNA, (A-A-G-C-G-C-A-U-C-U-U-C-U-A-C-U-U-C-A), complex with RNA (U-G-A-A-G-U-A-G-A-A-G-A-U-G-C-G-C-U-U) (1:1), sodium salt
RNA, (A-A-G-C-G-C-A-U-C-U-U-C-U-A-C-U-U-C-A), complex with RNA (U-G-A-A-G-U-A-G-A-A-G-A-U-G-C-G-C-U-U) (1:1), sodium salt
