Lumasiran (ALN-G01) is an siRNA compound that reduces hepatic oxalate production by targeting glycolate oxidase. Lumasiran inhibits the synthesis of oxalate, a toxic metabolite directly associated with the manifestations of primary hyperoxaluria type 1 (PH1), by silencing the gene encoding glycolate oxidase and depleting glycolate oxidase.

Physicochemical Properties

CAS #	1834610-13-7
Appearance	White to off-white solid powder
HS Tariff Code	2934.99.9001
Storage	Powder-20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment (e.g. under nitrogen), avoid exposure to moisture.
Shipping Condition	Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)

Biological Activity

ln Vitro	The cause of progressive renal injury in PH1 is urinary oxalate excretion, which is decreased by lumasiran [1].
ln Vivo	Lumasiran is an RNA interference (RNAi) twin that is targeted subcutaneously [2].
References	[1]. Lumasiran: First Approval. Drugs. 2021;81(2):277-282. [2]. Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 2021;384(13):1216-1226.

Solubility Data

Solubility (In Vitro)	H2O : ~100 mg/mL
Solubility (In Vivo)	Solubility in Formulation 1: 50 mg/mL (Infinity mM) in PBS (add these co-solvents sequentially from left to right, and one by one), clear solution; with sonication.  (Please use freshly prepared in vivo formulations for optimal results.)