| Field | Specification |
|---|---|
| Alternative names | ALN-AS1 |
| CAS no. | |
| Applications | |
| Molecular weight | |
| Purity | |
| Form | Solid |
| Storage | |
| Shipping | |
| Catalog no. (Mfr.) | |
| Main SKU |
Compound Overview
Givosiran, also known as ALN-AS1, is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. It downregulates ALAS1 mRNA, preventing accumulation of neurotoxic δ-aminolevulinic acid (ALA) and porphobilinogen (PBG), and demonstrates potent inhibitory activity against ALAS1 in mouse, rat and cynomolgus monkey models. It can be used in research on acute hepatic porphyria (AHP)[1][2]. It is supplied as a white to off-white solid at 94.21% purity (MW 16300.60).
Physical & Chemical Properties
| CAS Number | 1639325-43-1 |
|---|---|
| Molecular Weight | 16300.60 g/mol |
| Purity | 94.21% |
| Appearance | Solid |
| Color | White to off-white |
| 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) |
| Signaling Pathway | Epigenetics |
| Storage | -20°C, sealed storage, away from moisture. In solvent: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). |
| Shipping | Room temperature in continental US; may vary elsewhere. |
Literature Cited
Sources cited in this description and in the In Vitro & In Vivo Data tab. Peer-reviewed publications that used this product are listed under References.
[1]. Scott LJ. Givosiran: First Approval. Drugs. 2020 Feb;80(3):335-339.
Safety
For Research Use Only. Not for use in diagnostic or therapeutic procedures, and not for human or veterinary use. Handle in accordance with the Safety Data Sheet and your institution's chemical hygiene plan.
In Vivo
Serum and liver tissue ALAS1 mRNA levels in rats are effectively reduced by Givosiran (10 mg/kg, s.c., single dose), and the inhibition has a rapid onset and is long-lasting[2]. Givosiran (3 mg/kg, s.c., twice weekly for 3 weeks) fully prevents disease in the rat acute intermittent porphyria (AIP) model[2]. In mice, Givosiran (20 mg/kg, s.c., single dose) acts faster and inhibits more strongly than standard Hemoglobin treatment[2]. In cynomolgus monkey, Givosiran (1-10 mg/kg, s.c., as a single dose or once daily over 8 weeks) lowers serum and urinary ALAS1 levels; repeated dosing gives sustained inhibition that reverses once the drug is discontinued[2].
| 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. |
Data provided by the manufacturer. Numbered citations refer to the Literature Cited list in the product description.
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