1639325-44-2
Chemical Structure
Givosiran sodium
Synonym(s): ALN-AS1 sodium
- CAS No.: 1639325-44-2
- Formula:C524H650F16N173Na44O316P43S6
- Molecular Weight:16300.60 (free acid)
SMILES: [Givosiran (sodium)]
Biological Activity: Givosiran (ALN-AS1) sodium is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran sodium downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid (ALA) and porphobilinogen (PBG) levels. Givosiran sodium demonstrates potent inhibitory activity against ALAS1 in mouse, rat, and cynomolgus monkey models. Givosiran sodium can be used for the research of acute hepatic porphyria (AHP)[1][2].
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Givosiran sodium | 98.51% | Givosiran (ALN-AS1) sodium is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran sodium downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid (ALA) and porphobilinogen (PBG) levels. Givosiran sodium demonstrates potent inhibitory activity against ALAS1 in mouse, rat, and cynomolgus monkey models. Givosiran sodium can be used for the research of acute hepatic porphyria (AHP). | ||||||||||||||||||||
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- [1]. Scott LJ. Givosiran: First Approval. Drugs. 2020 Feb;80(3):335-339. [Content Brief]
- [2]. Chan A, et al. Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification. Mol Ther Nucleic Acids. 2015 Nov 3;4(11):e263. [Content Brief]
Keywords
