Givosiran is a small interfering RNA (siRNA) directed towards 5-aminolevulinic acid synthase, a critical enzyme in the heme biosynthesis pathway.[L10202] It is manufactured by Alnylam Pharmaceuticals and was first approved for use in the United States in Nove…

Patisiran is a first in class short interfering RNA for the treatment of patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis [L4220]. It is marketed as Onpattro which is formulated as patisiran within a liposome envelope for b…

Vutrisiran is a double-stranded small interfering ribonucleic acid (siRNA) that targets wild-type and mutant transthyretin (TTR) messenger RNA (mRNA).[L42065] This siRNA therapeutic is indicated for the treatment of neuropathies associated with hereditary tra…