| Description | Lumasiran sodium, an investigational RNA interference (RNAi) therapeutic agent, targets glycolate oxidase to reduce hepatic oxalate production, thereby reducing urinary oxalate excretion. This compound shows promise in addressing the underlying cause of progressive kidney failure in individuals with primary hyperoxaluria type 1 (PH1). |
| In vitro | 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]. |
| In vivo | Lumasiran sodium is a subcutaneously administered, liver-directed RNA interference (RNAi) therapeutic agent[2]. |
| molecular weight | N/A |
| CAS | 1834612-06-4 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
| References | 1. Scott LJ, et al. Lumasiran: First Approval. Drugs. 2021;81(2):277-282. 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. |