CAS 2175009-08-0 Fazirsiran - RNA / BOC Sciences

Fazirsiran

Fazirsiran is a second-generation RNAi drug. Fazirsiran consistes of a cholesterol-conjugated RNAi trigger (chol-RNAi) to selectively degrade AAT mRNA by RNAi and a melittin-derived peptide conjugated to N-acetylgalactosamine (NAG) formulated as the excipient EX1 to promote endosomal escape of the chol-RNAi in hepatocytes.

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Catalog

BRP-02075

Synonyms

ARO-AAT; RNA, ([1'-de(6-amino-9H-purin-9-yl)]dA-(5'→5')-sp-Am-Gm-Cm-Gm-Um-Um-Um-Am-(2'-deoxy-2'-fluoro)G-(2'-deoxy-2'-fluoro)G-(2'-deoxy-2'-fluoro)C-Am-Um-Gm-Um-Um-Um-Am-Am-Cm-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)G-sp-Um-(2'-deoxy-2'-fluoro)U-Am-(2'-deoxy-2'-fluoro)A-Am-Cm-Am-Um-Gm-(2'-deoxy-2'-fluoro)C-Cm-(2'-deoxy-2'-fluoro)U-Am-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy-2'-fluoro)C-Gm-(2'-deoxy-2'-fluoro)C-sp-Um) (1:1)

CAS

2175009-08-0

Appearance

White to off-white solid

Storage

Store at -20 °C, sealed storage, away from moisture

Sequence

RNA, (xagcguuuaggcauguuuaacax), complex with RNA (uguuaaacaugccuaaacgcu) (1:1)

Related CAS

2175009-09-1 (sodium salt)

Fazirsiran - CAS 2175009-08-0

Fazirsiran