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Synonyms: DCR-PHXC | NN-7022 | Rivfloza®
nedosiran is an approved drug (FDA (2023))
Compound class:
Nucleic acid
Comment: Nedosiran (DCR-PHXC) is a synthetic double-stranded siRNA drug. It contains a target-selective guide strand, plus a passenger strand that is attached to an extended nucleotide region that acts as a dicer substrate siRNA which is conjugated with four N-acetylgalactosamine (GalNAc) sugars. These modifications act to both stabilise the siRNA and optimise orientation for presentation to hepatocytes [2,4]. Nedosiran targets lactate dehydrogenase A (LDHA) mRNA as a mecahnism to reduce urinary oxalate levels as a treatment for primary hyperoxaluria.
We have been unable to locate a full chemical SMILES for the drug, or its HELM notation. |
