| Chemical Properties | Back Directory | [storage temp. ]
Store at -20°C | [form ]
Solid | [color ]
White to off-white | [Sequence]
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) |
| Hazard Information | Back Directory | [Uses]
Lumasiran (ALN-G01), a siRNA product, reduces hepatic oxalate production by targeting glycolate oxidase. By silencing the gene encoding glycolate oxidase, Lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of Primary hyperoxaluria type 1 (PH1)[1][2]. | [in vivo]
Lumasiran is a subcutaneously administered, liver-directed RNA interference (RNAi) therapeutic agent[2]. | [storage]
Store at -20°C | [References]
[1] Scott LJ, et al. Lumasiran: First Approval. Drugs. 2021;81(2):277-282. DOI:10.1007/s40265-020-01463-0
[2] Garrelfs SF, et al. Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 2021;384(13):1216-1226. DOI:10.1056/NEJMoa2021712 |
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| Company Name: |
Apextide Co Ltd
|
| Tel: |
15300650552; 15300650552 |
| Website: |
http://www.apextide.com |
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