Lumasiran sodium 

Lumasiran sodium, an investigational RNA interference (RNAi) therapeutic agent, reduces hepatic oxalate production by targeting glycolate oxidase. Lumasiran sodium reduces urinary oxalate excretion, the cause of progressive kidney failure in primary hyperoxaluria type 1 (PH1) ^[1][2].

Lumasiran sodium can be used for the research of PH1. By silencing the gene encoding glycolate oxidase, Lumasiran sodium depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of PH1^[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Lumasiran sodium is a subcutaneously administered, liver-directed RNA interference (RNAi) therapeutic agent^[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

17307.71

1834612-06-4

Solid

White to off-white

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

[Lumasiran (sodium)]

Room temperature in continental US; may vary elsewhere.

-20°C, stored under nitrogen, away from moisture

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

• [1]. Scott LJ, et al. Lumasiran: First Approval. Drugs. 2021;81(2):277-282.  [Content Brief]

  [2]. Garrelfs SF, et al. Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 2021 Apr 1;384(13):1216-1226.  [Content Brief]