1639325-43-1

1639325-43-1 structure

Name: Givosiran
Chemical Name: 21-[(2-Acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-N-[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]-6-[(3-{[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]amino}-3-oxopropoxy)methyl]-6-({12-[(2R,4R)-4-hydroxy-2-methyl-1-pyrrolidinyl]-12-oxododecanoyl}amino)-11,17-dioxo-4,8-dioxa-12,16-diazahenicosan-1-amide
CAS Number: 1639325-43-1
Molecular Formula: C₇₈H₁₃₉N₁₁O₃₀
Molecular Weight: 1710.994
Catalog: Research Areas Neurological Disease
Create Date: 2022-01-23 15:39:46
Modify Date: 2025-08-27 18:00:19
Givosiran (ALN-AS1) is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels. Givosiran can be used for the research of acute intermittent porphyria[1][2].

Name	21-[(2-Acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-N-[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]-6-[(3-{[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]amino}-3-oxopropoxy)methyl]-6-({12-[(2R,4R)-4-hydroxy-2-methyl-1-pyrrolidinyl]-12-oxododecanoyl}amino)-11,17-dioxo-4,8-dioxa-12,16-diazahenicosan-1-amide
Synonyms	21-[(2-Acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-N-[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]-6-[(3-{[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy ]pentanoyl}amino)propyl]amino}-3-oxopropoxy)methyl]-6-({12-[(2R,4R)-4-hydroxy-2-methyl-1-pyrrolidinyl]-12-oxododecanoyl}amino)-11,17-dioxo-4,8-dioxa-12,16-diazahenicosan-1-amide 1-Pyrrolidinedodecanamide, N-[2-[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]-1,1-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-gala ctopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]ethyl]-4-hydroxy-2-methyl-λ-oxo-, (2R,4R)-

Description	Givosiran (ALN-AS1) is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels. Givosiran can be used for the research of acute intermittent porphyria[1][2].
Related Catalog	Signaling Pathways >> Others >> Others Research Areas >> Neurological Disease
In Vitro	Givosiran is conjugated to a trivalent N-acetylgalactosamine ligand, which binds specifically to the asialoglycoprotein receptor, enabling targeted delivery to hepatocytes[2].
References	[1]. Scott LJ. Givosiran: First Approval. Drugs. 2020 Feb;80(3):335-339. [2]. Balwani M, et, al. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. N Engl J Med. 2020 Jun 11;382(24):2289-2301.