Givosiran
Based on 1 publication(s) in Google Scholar
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 (ALA) and porphobilinogen (PBG) levels. Givosiran demonstrates potent inhibitory activity against ALAS1 in mouse, rat, and cynomolgus monkey models. Givosiran can be used for the research of acute hepatic porphyria (AHP).
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- Pureza: 94.21%
- No. CAS: 1639325-43-1
- Peso molecular:16300.60
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Almacenamiento:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
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Actividad biológica
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Chemical Information
- Pureza y Documentación
- Referencias
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Publications Citing Use of MedChemExpress (MCE) Givosiran
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Actividad biológica
Givosiran (3 mg/kg, s.c., twice weekly for 3 weeks) exhibits complete preventive effect in the rat acute intermittent porphyria (AIP) model[2].
Givosiran (20 mg/kg, s.c., single dose) works faster and has stronger inhibition than standard Hemoglobin (HY-P2995) treatment in mice[2].
Givosiran (1-10 mg/kg, s.c., single dose or once daily for 8 weeks) reduces serum and urinary ALAS1 levels in cynomolgus monkey, and repeated administration results in sustained inhibition that is reversible after drug discontinuation[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:AIP model induced by Phenobarbital established in SD rats[2]
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Dosage:3 mg/kg
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Administration:Subcutaneous injection (s.c.), twice weekly for 3 weeks
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Result:Completely prevented inducible ALAS1 elevation and associated metabolite accumulation.
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Animal Model:AIP model induced by Phenobarbital established in T1/T2 AIP mice[2]
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Dosage:20 mg/kg
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Administration:Subcutaneous injection (s.c.), single dose
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Result:Inhibited ALAS1 faster and stronger than Hemoglobin.
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Animal Model:AIP model induced by Phenobarbital established in cynomolgus monkey (M. fascicularis)[2]
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Dosage:1 and 10 mg/kg (single dose); 5 mg/kg (8 weeks)
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Administration:Subcutaneous injection (s.c.), single dose or once daily for 8 weeks
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Result:Dose-dependently and reversibly inhibited ALAS1 levels and repeated dosing achieved sustained suppression.
Chemical Information
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No. CAS 1639325-43-1
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Appearance Solid
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Peso molecular 16300.60
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Color White to off-white
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SMILES
[Givosiran]
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Synonyms
ALN-AS1
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Sequence
RNA, (Cm-sp-Am-sp-Gm-Am-Am-Am-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)C-Am-Um-Cm-Um-Um-Am), 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]- 14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]-1,12,19,25-tetraoxo-16-oxa13,20,24-triazanonacos-1-yl]-4-hydroxy-2-pyrrolidinyl]methyl hydrogen phosphate], complex with RNA (Um-sp-(2'-deoxy-2'-fluoro)A-sp-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)U-Gm-sp-Gm-sp-Um) (1:1)
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Envío
Room temperature in continental US; may vary elsewhere.
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Almacenamiento
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Publications (1)
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Journal Impact Factor
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Most Recent
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Am J Reprod Immunol
Active Heme Metabolism Suppresses Macrophage Phagocytosis via the TLR4/Type I IFN Signaling/CD36 in Uterine Endometrial Cancer. [Abstract]2024 Aug;92(2):e13916. PMID: 39166450
Givosiran purchased from MedChemExpress. Usage Cited in: Am J Reprod Immunol. 2024 Aug;92(2):e13916. [Abstract]
Heme concentrations in the RL95-2 cells treated by succinate 2.5 mM for 24 h, then the supplement of 5 µM SDHB inhibitor (Fluopyam) or 1 µM ALAS1 inhibitor (Givosiran) for 24 h.
Pureza y Documentación
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Ficha de datos (277 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Portuguese - PT (252 KB)
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Instrucciones de manejo (2242 KB)
Referencias
[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]
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
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