MGX-001

MGX-001 is an investigational gene-editing therapy being developed by Metagenomi for the treatment of hemophilia A. The therapy is designed to provide a one-time, potentially curative treatment by integrating a functional version of the F8 gene—which encodes factor VIII (FVIII), the clotting protein deficient in hemophilia A—into a specific site within the albumin gene in liver cells. This integration is achieved using a combination of adeno-associated virus (AAV) vectors to deliver the F8 gene and lipid nanoparticles containing guide RNA and mRNA encoding a nuclease (MG29-1). The guide RNA directs the nuclease to cut at a precise location in the albumin gene, enabling targeted insertion of F8. Preclinical studies in nonhuman primates have demonstrated sustained FVIII activity for over 12–16 months with promising safety and durability profiles. As of early 2025, MGX-001 remains in preclinical development with IND-enabling activities underway and plans for clinical trials targeting both adults and children with hemophilia A[1][2][3][4][5][6][7].