RN679

RN679 is a liver-targeted small interfering RNA (siRNA) candidate developed by Shanghai Rona Therapeutics for the treatment of Acute Hepatic Porphyria (AHP). Developed using the proprietary GAIA platform, RN679 is a GalNAc-conjugated siRNA designed for highly specific delivery to hepatocytes via the asialoglycoprotein receptor (ASGPR). The therapeutic mechanism involves the targeted degradation of mRNA encoding delta-aminolevulinate synthase 1 (ALAS1), the rate-limiting enzyme in the hepatic heme biosynthesis pathway. By silencing ALAS1, RN679 prevents the overproduction and accumulation of neurotoxic heme precursors, such as delta-aminolevulinic acid (ALA) and porphobilinogen (PBG), which are the primary drivers of acute porphyria attacks and associated neurological symptoms. As of early 2024, RN679 is in the preclinical stage of development.