pGX9436

pGX9436 is a DNA-encoded therapeutic protein (DPROT) candidate developed by Inovio Pharmaceuticals for the treatment of hemophilia A. It consists of a plasmid DNA construct encoding a human Factor VIII (FVIII) protein, optimized for enhanced stability, expression, and secretion from skeletal muscle cells. The therapy is delivered via intramuscular injection followed by electroporation using the CELLECTRA™ device, which facilitates the uptake of the plasmid by muscle cells. Once inside, the muscle cells serve as a long-term biofactory for the sustained production and systemic secretion of functional FVIII. This non-viral gene therapy approach aims to provide durable therapeutic levels of clotting factor and allows for redosing, potentially overcoming limitations associated with viral-vectored gene therapies such as hepatotoxicity and pre-existing immunity.