Fig. 1 · Structure

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Givosiran

ALAS1 Inhibitor

Givosiran is a small interfering RNA that specifically inhibits hepatic aminolevulinate synthase 1 (ALAS1) by targeting its messenger RNA. This inhibition leads to decreased levels of the neurotoxic compounds δ-aminolevulinic acid (ALA) and porphobilinogen (PBG). Givosiran exhibits significant efficacy in reducing ALAS1 activity in various preclinical models, including mouse, rat, and cynomolgus monkey studies. This reagent is valuable for research focused on acute hepatic porphyria (AHP) and its associated metabolic disturbances.

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Specifications

Manufacturer Adooq Bioscience
Actions Inhibitor
CAS Number 1639325-43-1
Applications ALAS1 Inhibitor
Molecular Weight 16300.60
Synonyms ALN-AS1

Storage & Solubility

In Vitro Solubility

Reported Solubility

No reported solubility data are available for this product.

Preparing Stock Solutions

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.

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