Givosiran

Givosiran (ALN-AS1) is a small interfering RNA compound that selectively targets hepatic aminolevulinate synthase 1 (ALAS1) mRNA, leading to efficient downregulation of ALAS1 expression and consequent inhibition of neurotoxic δ-aminolevulinic acid and porphobilinogen accumulation. Givosiran provides a mechanism-driven research tool with significant relevance for studying metabolic dysregulation and therapeutic intervention strategies in acute intermittent porphyria.