The New England Journal of Medicine

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter.
Healio

Gene therapy increases factor VIII activity, reduces bleeding in severe hemophilia A

Please provide your email address to receive an email when new articles are posted on . Valoctocogene roxaparvovec induced endogenous factor VIII production and significantly reduced bleeding and ...
The American Journal of Managed Care

Hemophilia A Gene Therapy Reduced Bleeding Events, Need for Clotting Factors

The phase 3 trial of valoctocogene roxaparvovec for severe hemophilia A showed it reduced bleeding events as well as the use of factor VIII concentrates. A phase 3 trial of valoctocogene roxaparvovec, ...
Nature

AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice

Hemophilia A gene therapy has been hampered by immune responses to vector-associated antigens and by neutralizing antibodies or inhibitors against the factor VIII (FVIII) protein; these ‘inhibitors’ ...