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].
CAS Number
[1639325-43-1]
Molecular Formula
C524H694F16N173O316P43S6
Molecular Weight
16300.6
Smiles
[Givosiran]
Target
Small Interfering RNA (siRNA)
Purity
98.51
Solubility
10 mM in H2O
Storage Conditions
-20°C (Powder, sealed storage, away from moisture)