Fazirsiran (ARO-AAT) is a second-generationRNAidrug. 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^[1].

Fazirsiran (ARO-AAT; sc; 4 mg/kg; on 1, 15, 29, 43 days during 57 days) deeply reduces Z-AAT mRNA and protein in five-week-old male PiZ mice^[1].

15587.60

2175009-08-0

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)

Room temperature in continental US; may vary elsewhere.

Please store the product under the recommended conditions in the Certificate of Analysis.