mRNA-3927

mRNA-3927 is an investigational, intravenous, lipid nanoparticle (LNP)-encapsulated dual mRNA therapy developed by Moderna for the treatment of propionic acidemia. It consists of two messenger RNAs encoding the alpha (PCCA) and beta (PCCB) subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC). By delivering these mRNAs to liver cells, mRNA-3927 enables intracellular production of functional PCC enzyme, aiming to restore or replace deficient or dysfunctional enzyme activity in patients with propionic acidemia. The therapy has shown encouraging early clinical results in reducing metabolic decompensation events and has been generally well tolerated in Phase 1/2 trials[1][2][4][5][6][7][8].

Other names

modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated in lipid nanoparticleslipid nanoparticle-encapsulated dual mRNA therapy encoding PCCA and PCCBmodified mRNA encoding human PCCA and PCCB encapsulated in lipid nanoparticles