Givosiran sodium (Synonyms: ALN-AS1 sodium)

Name: Givosiran sodium
Brand: MedChemExpress
SKU: HY-132610A
Rating: 4.5 (1 reviews)

Cat. No.: HY-132610A Purity: 98.51%: Data Sheet
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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).

For research use only. We do not sell to patients.

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-oxa-13,20,24-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), sodium salt (1:1:43)

CAS No. : 1639325-44-2

Size	Stock	Quantity
1 mg	In-stock	Estimated Time of Arrival: December 31
5 mg	In-stock	Estimated Time of Arrival: December 31
10 mg	In-stock	Estimated Time of Arrival: December 31
25 mg	In-stock	Estimated Time of Arrival: December 31
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Based on 1 publication(s) in Google Scholar

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Givosiran In-stock

1 Publications Citing Use of MCE Givosiran sodium

•Am J Reprod Immunol. 2024 Aug;92(2):e13916. [Abstract]

Others

: Givosiran sodium 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.

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Description

In Vivo

Givosiran sodium (10 mg/kg, s.c., single dose) effectively reduces ALAS1 mRNA levels in liver tissue and serum in rats with rapid onset and long-lasting inhibitory properties^[2].
Givosiran sodium (3 mg/kg, s.c., twice weekly for 3 weeks) exhibits complete preventive effect in the rat acute intermittent porphyria (AIP) model^[2].
Givosiran sodium (20 mg/kg, s.c., single dose) works faster and has stronger inhibition than standard Hemoglobin (HY-P2995) treatment in mice^[2].
Givosiran sodium (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.

Animal Model:	AIP model induced by Phenobarbital established in SD rats^[2]
Dosage:	3 mg/kg
Administration:	Subcutaneous injection (s.c.), twice weekly for 3 weeks
Result:	Completely prevented inducible ALAS1 elevation and associated metabolite accumulation.

Animal Model:	AIP model induced by Phenobarbital established in T1/T2 AIP mice^[2]
Dosage:	20 mg/kg
Administration:	Subcutaneous injection (s.c.), single dose
Result:	Inhibited ALAS1 faster and stronger than Hemoglobin.

Animal Model:	AIP model induced by Phenobarbital established in cynomolgus monkey (M. fascicularis)^[2]
Dosage:	1 and 10 mg/kg (single dose); 5 mg/kg (8 weeks)
Administration:	Subcutaneous injection (s.c.), single dose or once daily for 8 weeks
Result:	Dose-dependently and reversibly inhibited ALAS1 levels and repeated dosing achieved sustained suppression.

Clinical Trial

Molecular Weight

16300.60 (free acid)

Formula

C₅₂₄H₆₅₀F₁₆N₁₇₃Na₄₄O₃₁₆P₄₃S₆

CAS No.

1639325-44-2

Appearance

Solid

Color

White to off-white

SMILES

[Givosiran (sodium)]

Shipping

Room temperature in continental US; may vary elsewhere.

Storage

-20°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

Purity & Documentation

Purity: 98.71%

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Data Sheet (271 KB) SDS (252 KB)

COA (249 KB) LCMS (410 KB)

Handling Instructions (2242 KB)

References

[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]

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Metabolic Disease

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Help & FAQs

Do most proteins show cross-species activity?
Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

Givosiran1639325-44-2ALN-AS1Small Interfering RNA (siRNA)siRNAaminolevulinateALAS1mRNAδ-aminolevulinicacidporphobilinogenacuteintermittentporphyriaInhibitorinhibitorinhibit

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Givosiran sodium (Synonyms: ALN-AS1 sodium)

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Other Forms of Givosiran sodium:

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1 Publications Citing Use of MCE Givosiran sodium

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