The US Food and Drug Administration (FDA) has approved the first gene therapy for adults with severe haemophilia A. BioMarin’s Roctavian (valoctocogene roxaparvovec-rvox) has been approved for the treatment of adults with severe haemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected […]