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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. |
| References |
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1. Baum MA, Langman C, Cochat P, Lieske JC, Moochhala SH, Hamamoto S, Satoh H, Mourani C, Ariceta G, Torres A et al.. (2023)
PHYOX2: a pivotal randomized study of nedosiran in primary hyperoxaluria type 1 or 2. Kidney Int, 103 (1): 207-217. [PMID:36007597] |
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2. Li Q, Dong M, Chen P. (2024)
Advances in structural-guided modifications of siRNA. Bioorg Med Chem, 110: 117825. [PMID:38954918] |
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3. Syed YY. (2023)
Nedosiran: First Approval. Drugs, 83 (18): 1729-1733. [PMID:38060091] |
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4. Traber GM, Yu AM. (2024)
The Growing Class of Novel RNAi Therapeutics. Mol Pharmacol, 106 (1): 13-20. [PMID:38719476] |
