HGD mRNA-LNP

HGD mRNA-LNP is an experimental liver-directed mRNA therapeutic designed for the treatment of Alkaptonuria (AKU), a rare autosomal recessive metabolic disorder. AKU is caused by deficiency of the enzyme homogentisate 1,2-dioxygenase (HGD), leading to the accumulation of homogentisic acid (HGA) and subsequent multi-systemic damage, including severe arthropathy. The therapeutic consists of mRNA encoding the human HGD protein encapsulated within lipid nanoparticles (LNPs) for targeted delivery to hepatocytes. Upon administration, the mRNA is translated into functional HGD enzyme, restoring the metabolic breakdown of HGA. Preclinical studies in AKU mouse models have demonstrated that intravenous injection of HGD mRNA-LNP can normalize plasma and urinary HGA levels in a dose-dependent manner.

Other names

HGD-mRNA-LNPsmRNA-LNPs encoding HGD