Fig. 1 · Structure
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Givosiran sodium
ALAS1 Inhibitor
Givosiran sodium is a small interfering RNA that specifically inhibits hepatic aminolevulinate synthase 1 (ALAS1) by targeting its messenger RNA. This downregulation leads to a significant reduction in the accumulation of neurotoxic metabolites, such as δ-aminolevulinic acid (ALA) and porphobilinogen (PBG). Givosiran sodium exhibits strong inhibitory effects on ALAS1 in preclinical models, including mouse, rat, and cynomolgus monkey studies. It is valuable for research focused on acute hepatic porphyria (AHP) and related disorders.
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Specifications
| Manufacturer | Adooq Bioscience |
|---|---|
| Actions | Inhibitor |
| CAS Number | 1639325-44-2 |
| Applications | ALAS1 Inhibitor |
| Formula | C524H650F16N173Na44O316P43S6 |
| Molecular Weight | 16300.60 (free acid) |
| Synonyms | ALN-AS1 sodium |
Storage & Solubility
In Vitro Solubility
Reported Solubility
No reported solubility data are available for this product.
Preparing Stock Solutions
| Concentration | |
|---|---|
Values use four significant digits. Prepare to the final solution volume; this is not the volume of solvent to add.
Stock preparation is unavailable for this product molecular weight.
Calculated stock concentrations do not establish solvent solubility. Refer to the reported solubility data for experimental guidance.
Storage
Store powder at -20°C, keep desiccated.
In powder form, the chemical is stable for 36 months.
In solution, store at -20°C and use within 1 month to prevent loss of potency. Aliquot to avoid multiple freeze/thaw cycles.