MRT5201

MRT5201 is an investigational mRNA-based therapeutic developed for the treatment of ornithine transcarbamylase (OTC) deficiency, a rare genetic disorder that leads to excessive ammonia accumulation in the blood. The drug works by delivering messenger RNA (mRNA) encoding a fully functional human OTC enzyme directly to the liver via intravenous administration. This enables hepatocytes (liver cells) to produce the normal OTC enzyme, compensating for the genetic deficiency and restoring urea cycle function. Preclinical studies demonstrated that a single IV dose of MRT5201 could induce durable expression of functional human OTC protein in mouse models, resulting in normalized ammonia and orotic acid levels for up to four weeks. The drug has been granted orphan drug designation in both the U.S. and EU for this indication[1][4][5][7].