INO-D001

INO-D001 is an investigational non-viral DNA medicine (DNA plasmid) developed by Inovio for the treatment of hemophilia A. It is designed using Inovio's DNA-encoded Protein (DPROT) technology platform. INO-D001 consists of a synthetic DNA plasmid encoding human Coagulation Factor VIII (FVIII) with an IgE leader sequence, a partial B domain, and specific modifications (such as the R571S gain-of-function mutation) to optimize expression and clotting activity. Delivered intramuscularly followed by electroporation using Inovio's CELLECTRA device, INO-D001 enables host muscle cells to produce functional FVIII protein in vivo, offering a potential next-generation protein replacement therapy to correct the bleeding phenotype in hemophilia A patients.

Other names

DNA-encoded FVIIIDNA-encoded Factor VIIIDPROT-FVIII