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nedosiran
- Development stage
- Approved
- Lead developer
- Novo Nordisk
- 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
Nedosiran is a small interfering RNA (siRNA) therapeutic used to treat primary hyperoxaluria type 1 (PH1), a rare inherited disorder characterized by excessive oxalate production that can lead to kidney stones and renal failure. Nedosiran works by specifically targeting and degrading the messenger RNA (mRNA) of hepatic lactate dehydrogenase A (LDHA), an enzyme responsible for converting glyoxylate to oxalate in the liver. By reducing LDHA levels, nedosiran lowers hepatic oxalate production, thereby decreasing urinary and systemic oxalate burden. The drug is administered as a subcutaneous injection and is conjugated with GalNAc sugars for targeted delivery to hepatocytes via asialoglycoprotein receptors. Nedosiran was developed by Dicerna Pharmaceuticals and is marketed under the brand name Rivfloza[2][3][4][6][7].
- Brand names
- Rivfloza
- Other names
- nedosiran sodium
02
Targets
Lactate dehydrogenase A messenger RNA (LDHA mRNA)
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