mRNA-3905

mRNA-3905 is an investigational mRNA-based therapeutic developed by Moderna for the treatment of acute intermittent porphyria (AIP). It consists of human porphobilinogen deaminase (hPBGD) mRNA, encoded by the *HMBS* gene, encapsulated within lipid nanoparticles (LNPs) for systemic delivery to the liver. AIP is a rare metabolic disorder caused by a deficiency in the PBGD enzyme, which leads to the accumulation of neurotoxic porphyrin precursors like aminolevulinic acid (ALA) and porphobilinogen (PBG). mRNA-3905 is designed to restore intracellular PBGD enzyme activity in hepatocytes, thereby normalizing the heme biosynthesis pathway and preventing the acute neurovisceral attacks associated with the disease. Preclinical data demonstrated that the therapy can rapidly reduce porphyrin levels and protect against symptoms such as hypertension and motor impairment.

Other names

human porphobilinogen deaminase messenger RNAhPBGD mRNAhuman PBGD mRNA