citrin mRNA

**citrin mRNA** is an investigational **mRNA-based drug** being developed by **Moderna** for **citrin deficiency**, including **adult-onset type II citrullinemia**, **neonatal intrahepatic cholestasis caused by citrin deficiency**, and related metabolic manifestations. The therapy consists of **codon-optimized mRNA encoding human citrin**, formulated in **lipid nanoparticles** for hepatic delivery, with the goal of restoring expression of the liver-specific mitochondrial aspartate glutamate carrier encoded by **SLC25A13**. By enabling production of functional citrin protein in hepatocytes, the drug is intended to correct the underlying transporter deficiency and improve downstream metabolic abnormalities such as hypercitrullinemia and hyperammonemia. Available context supports a **preclinical or early development-stage** program, and no specific brand name was identified.

Other names

citrin mRNAcitrin mRNA-LNP