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Synonyms: ALN-AS1 | Givlaari®
givosiran is an approved drug (FDA (2019))
Compound class:
Nucleic acid
Comment: Givosiran (ALN-AS1) is a double-stranded N-acetylgalactosamine (GalNAc)-conjugated small interfering ribonucleic acid (siRNA) drug. It inhibits hepatic synthesis of 5'-aminolevulinate synthase 1 as a mechanism to reduce accumulation of toxic heme intermediates in patients with acute hepatic porphyria (which arises from deficient aminolevulinic acid dehydratase and porphobilinogen deaminase activities). GalNAc-conjugation is a strategy used to promote delivery of the drug to the liver [1]- this is annotated as 'R1' in the sequence provided here. We have been unable to locate a full chemical SMILES for the drug, or its HELM notation.
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