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 New England Journal of Medicine

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Valoctocogene roxaparvovec delivers a B-domain–deleted factor VIII coding sequence with an adeno-associated virus vector to prevent bleeding in persons with severe hemophilia A. The findings of a ...
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, ...
Medscape

'Promise of a Cure': Gene Therapy for Hemophilia A

ATLANTA — Patients with hemophilia A who received a single infusion of an investigational gene therapy called valoctocogene roxaparvovec showed substantially increased levels of the essential blood ...