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).
In Vitro
Lumasiran reduced urinary oxalate excretion, the cause of progressive kidney failure in PH1. MCE has not independently confirmed the accuracy of these methods. They are for reference only.
In Vivo
Lumasiran is a subcutaneously administered, liver-directed RNA interference (RNAi) therapeutic agent. MCE has not independently confirmed the accuracy of these methods. They are for reference only.
