ARLN and acute intermittent porphyria: Some of the novel RNAi therapeutics in the clinical development pipeline for SRT, all targeting liver diseases, include: ALN-AS1 (Givosiran) silencing the haem biosynthesis gene ALAS1 as treatment for acute intermittent porphyria (MIM 176000), currently under Phase III trial [87]; and ALN-GO1 (Lumasiran) silencing HAO1, having completed Phase II [65], as well as DCR-PHXC (Phase I) silencing the LDH gene [68], as treatment for PH1.