Valoctocogene roxaparvovec (AAV5-hFVIII-SQ; proposed name: Valrox) is a single-administration hemophilia A gene therapy that combines a viral capsid (adeno-associated virus type 5 [AAV5]) with a factor VIII (FVIII) gene. The therapeutic viral vector delivers FVIII DNA to patient cells, where it can be expressed as FVIII protein. Valoctocogene roxaparvovec has the potential to eliminate the need for frequent, repeated prophylactic infusions.

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