Givosiran (ALN-AS1) sodium是一种靶向肝脏氨基乙酰丙酸合酶1（ALAS1）信使RNA的小干扰RNA。它通过下调ALAS1 mRNA,进而抑制神经毒性δ-氨基乙酰丙酸（ALA）和胆色素原（PBG）的积累。在针对小鼠、大鼠和食蟹猴的模型中,Givosiran sodium均显示出对ALAS1的强效抑制能力。该化合物可用于急性肝性卟啉症（AHP）的相关研究。

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