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lumasiran
- Development stage
- Approved
- Lead developer
- Alnylam Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Lumasiran is a synthetic double-stranded small interfering RNA (siRNA) drug used for the treatment of primary hyperoxaluria type 1 (PH1), a rare genetic disorder characterized by excessive oxalate production that can lead to kidney stones and renal failure. Lumasiran works by targeting the hydroxyacid oxidase 1 (HAO1) gene messenger RNA in hepatocytes through RNA interference, thereby reducing the production of glycolate oxidase (GO). This reduction in GO decreases glyoxylate and subsequently lowers oxalate synthesis. The drug is administered as a subcutaneous injection and is approved for use in both pediatric and adult patients.
- Brand names
- Oxlumo
- Other names
- lumasiran sodium
02
Targets
HAO1 (Glycolate Oxidase)
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