mRNA-3705

mRNA-3705 is an investigational mRNA-based therapeutic developed by Moderna for the treatment of methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MUT) deficiency. The drug consists of messenger RNA encoding the human MUT enzyme, encapsulated within Moderna's proprietary lipid nanoparticle (LNP) delivery system. By delivering these genetic instructions, mRNA-3705 enables cells to produce functional MUT enzyme, addressing the underlying cause of MMA—a rare genetic disorder characterized by an inability to properly break down certain proteins and fats, leading to toxic metabolite accumulation. The therapy is administered via intravenous infusion and has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the U.S. FDA[1][2][6].

Other names

CX-020629CX020629CX 020629