Givosiran

Givosiran (ALN-AS1) is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid (ALA) and porphobilinogen (PBG) levels. Givosiran demonstrates potent inhibitory activity against ALAS1 in mouse, rat, and cynomolgus monkey models. Givosiran can be used for the research of acute hepatic porphyria (AHP) [1][2].

Product Specifications

CAS Number

[1639325-43-1]

Product Name Alternative

ALN-AS1

UNSPSC

12352208

Target

Small Interfering RNA (siRNA)

Type

Oligonucleotides

Related Pathways

Epigenetics

Applications

Neuroscience-Neuromodulation

Field of Research

Metabolic Disease

Assay Protocol

https://www.medchemexpress.com/givosiran.html

Purity

98.51

Solubility

10 mM in H2O

Smiles

[Givosiran]

Molecular Formula

C524H694F16N173O316P43S6

Molecular Weight

16300.60

References & Citations

[1]Scott LJ. Givosiran: First Approval. Drugs. 2020 Feb;80 (3) :335-339.|[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.