siRNAs formulated as a lipid complex or covalently linked to specific ligands enabled FDA approval of ONPATTRO® (patisiran) for treatment of polyneuropathy in patients with hereditary transthyretin-mediated amyloidosis and GIVLAARITM (givosiran) used for treatment of acute hepatic porphyria (AHP) [64,65]. This evidence concerns the gene TTR and Acute hepatic porphyria.