TTR and Acute hepatic porphyria: Inotersen (antisense) and Patisirian (first ever siRNA approved by FDA) are both targeting transthyretin (TTR) for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis [6,7], while Givlaari (world’s first-ever approved GalNAc-conjugate RNAi therapeutic) targets aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria [8].