PLC-mcoET3

PLC-mcoET3 is an investigational cell and gene therapy product consisting of human placenta-derived mesenchymal cells (placental cells, PLC) transduced with a lentiviral vector encoding mcoET3, a myeloid codon-optimized, bioengineered factor VIII (FVIII) transgene with enhanced expression and activity.[2][5] After transplantation, the modified PLC engraft in multiple organs, particularly the liver, and secrete bioactive ET3 FVIII into the circulation at sustained, therapeutically meaningful levels without significant toxicity or strong alloimmune responses in large-animal (sheep) models.[2][5][7] PLC-mcoET3 also constitutively produce endogenous human FVIII and von Willebrand factor (vWF), although physiological shear stress downregulates this endogenous production while maintaining stable expression of the therapeutic mcoET3 transgene.[1] The primary intended indication is long-term correction or amelioration of hemophilia A, including potential prenatal treatment via in utero transplantation.

Other names

ET3-secreting human placental cellsET-3-secreting human placental cellsET 3-secreting human placental cellsFVIII/ET3-secreting human placental cellshuman PLC transduced with mcoET3