ARCT-810

ARCT-810 is an investigational mRNA replacement therapy developed to treat ornithine transcarbamylase (OTC) deficiency, a rare genetic disorder of the urea cycle. The drug delivers messenger RNA encoding the functional OTC enzyme directly to hepatocytes (liver cells), enabling patients with OTC deficiency to produce their own functional enzyme. This approach aims to restore normal urea cycle activity, reduce toxic ammonia accumulation, and improve clinical outcomes for affected individuals. ARCT-810 uses Arcturus Therapeutics' proprietary LUNAR lipid nanoparticle delivery platform and has received orphan drug and rare pediatric disease designations from regulatory agencies[1][2][3][4][5][6][8].