patisiran

Patisiran is a double-stranded small interfering RNA (siRNA) therapeutic formulated in a lipid nanoparticle for intravenous infusion. It is designed to treat the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults by targeting and degrading both mutant and wild-type transthyretin (TTR) mRNA through RNA interference, leading to reduced serum TTR protein levels and decreased TTR deposits in tissues. Developed by Alnylam Pharmaceuticals, it was the first FDA-approved siRNA drug, receiving approval in 2018 under the brand name Onpattro. Patisiran’s mechanism involves delivery to hepatocytes where it is processed by Dicer enzyme; one strand of the siRNA guides the RNA-induced silencing complex (RISC) to complementary TTR mRNA, resulting in its cleavage and silencing[1][3][4][5].

Brand names

Onpattro

Other names

patisiranum