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Givosiran

Name: Givosiran
Brand: TargetMol
SKU: T39123
Rating: 4.5 (1 reviews)

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Catalog No. T39123Cas No. 1639325-43-1

Alias ALN-AS1

Givosiran (ALN-AS1) is a small interfering RNA compound that selectively targets hepatic aminolevulinate synthase 1 (ALAS1) mRNA, leading to efficient downregulation of ALAS1 expression and consequent inhibition of neurotoxic δ-aminolevulinic acid and porphobilinogen accumulation. Givosiran provides a mechanism-driven research tool with significant relevance for studying metabolic dysregulation and therapeutic intervention strategies in acute intermittent porphyria.

Givosiran

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Catalog No. T39123Alias ALN-AS1Cas No. 1639325-43-1

Pack Size	Price	USA Warehouse	Global Warehouse
1 mg	$242	2-4 weeks	2-4 weeks
5 mg	$630	2-4 weeks	2-4 weeks
10 mg	$983	2-4 weeks	2-4 weeks
25 mg	$1,970	2-4 weeks	2-4 weeks
50 mg	$3,150	2-4 weeks	2-4 weeks
100 mg	$5,030	2-4 weeks	2-4 weeks

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In Stock Estimated shipping dateUSA Warehouse[1-2 days] Global Warehouse[5-7 days]

All TargetMol products are for research purposes only and cannot be used for human consumption. We do not provide products or services to individuals. Please comply with the intended use and do not use TargetMol products for any other purpose.

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Product Introduction

Bioactivity

Description	Givosiran (ALN-AS1) is a small interfering RNA compound that selectively targets hepatic aminolevulinate synthase 1 (ALAS1) mRNA, leading to efficient downregulation of ALAS1 expression and consequent inhibition of neurotoxic δ-aminolevulinic acid and porphobilinogen accumulation. Givosiran provides a mechanism-driven research tool with significant relevance for studying metabolic dysregulation and therapeutic intervention strategies in acute intermittent porphyria.
In vitro	Givosiran is conjugated to a trivalent N-acetylgalactosamine ligand for specific binding to the asialoglycoprotein receptor, facilitating targeted hepatocyte delivery[2].
In vivo	In rat models, a single subcutaneous dose of Givosiran (10 mg/kg) reduced ALAS1 mRNA levels in both liver tissue and serum. In a phenobarbital-induced Acute Intermittent Porphyria (AIP) rat model, a regimen of 3 mg/kg (twice weekly for 3 weeks) prevented the elevation of ALAS1 and the accumulation of toxic metabolites. In T1/T2 AIP mice, a single dose of Givosiran (20 mg/kg) inhibited ALAS1 more rapidly than Hemin treatment. Studies in cynomolgus monkeys (1-10 mg/kg) showed a dose-dependent reduction of ALAS1 in serum and urine, with effects being reversible after an 8-week dosing period [2].
Synonyms	ALN-AS1

Chemical Properties

Molecular Weight	16300.60
Cas No.	1639325-43-1
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)

Storage & Solubility Information

Storage

keep away from direct sunlight | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature.

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In Vivo Formulation Calculator (Clear solution)

Please enter your animal experiment information in the following box and click Calculate to obtain the stock solution preparation method and in vivo formula preparation method:

For example, if the intended dosage is 10 mg/kg for animals weighing 20 g , with a dosing volume of 100 μL per animal, TargetMol | Animal experiments

and a total of 10 animals are to be administered, using a formulation of TargetMol | reagent

10% DMSO+ 40% PEG300+ 5% Tween 80+ 45% Saline/PBS/ddH₂O , the resulting working solution concentration would be 2 mg/mL.

Stock Solution Preparation:

Dissolve 2 mg of the compound in 100 μL DMSO TargetMol | reagent to obtain a stock solution at a concentration of 20 mg/mL . If the required concentration exceeds the compound's known solubility, please contact us for technical support before proceeding.

Preparation of the In Vivo Formulation:

1) Add 100 μL of the DMSO TargetMol | reagent stock solution to 400 μL PEG300 and mix thoroughly until the solution becomes clear.

2) Add 50 μL Tween 80 and mix well until fully clarified.

3) Add 450 μL Saline,PBS or ddH₂O TargetMol | reagent and mix thoroughly until a homogeneous solution is obtained.

This example is provided solely to demonstrate the use of the In Vivo Formulation Calculator and does not constitute a recommended formulation for any specific compound. Please select an appropriate dissolution and formulation strategy based on your experimental model and route of administration.

All co-solvents required for this protocol, includingDMSO, PEG300/PEG400, Tween 80, SBE-β-CD, and Corn oil, are available for purchase on the TargetMol website.

Dose Conversion

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Tech Support

Please see Inhibitor Handling Instructions for more frequently ask questions. Topics include: how to prepare stock solutions, how to store products, and cautions on cell-based assays & animal experiments, etc

Keywords

Givosiran