Givosiran

Names

[ Name ]:
Givosiran

[Synonym ]:
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)-

Biological Activity

[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.

Chemical & Physical Properties

[ Density]:
1.3±0.1 g/cm3

[ Boiling Point ]:
1780.6±65.0 °C at 760 mmHg

[ Molecular Formula ]:
C₇₈H₁₃₉N₁₁O₃₀

[ Molecular Weight ]:
1710.994

[ Flash Point ]:
1030.6±34.3 °C

[ Exact Mass ]:
1709.968872

[ LogP ]:
-4.24

[ Vapour Pressure ]:
0.0±0.6 mmHg at 25°C

[ Index of Refraction ]:
1.579

Related Compounds

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