Hemophilia A and B are rare X-linked bleeding disorders caused by mutations in the genes
encoding coagulation factor VIII (FVIII) and factor IX (FIX). Hemophilia A (HA) is more …

Background Moderate-to-severe hemophilia B is treated with lifelong, continuous
coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims …

Background Valoctocogene roxaparvovec delivers a B-domain–deleted factor VIII coding
sequence with an adeno-associated virus vector to prevent bleeding in persons with severe …

Progress in hemophilia therapy has been remarkable in the first 20 years of the third
millennium, but the innovation began with the description the fractionation of plasma in …

Background Clinical trial data are scarce for the use of prophylaxis in people with non-
severe haemophilia A. The HAVEN 6 study aims to assess safety and efficacy of …

Background Factor VIII replacement products have improved the care of patients with
hemophilia A, but the short half-life of these products affects the patients' quality of life. The …

Etranacogene dezaparvovec (AMT-061) is a recombinant AAV5 vector including a gene
cassette containing the factor IX (FIX) Padua variant under the control of a liver-specific …

Factor VIII (FVIII) replacement products enable comprehensive care in hemophilia A.
Treatment goals in severe hemophilia A are expanding beyond low annualized bleed rates …

The mainstay of hemophilia treatment is to prevent bleeding through regular long-term
prophylaxis and to control acute breakthrough bleeds. Various treatment options are …

Gene therapy has the potential to overcome many of the limitations of prophylactic
hemophilia therapies. Several clinical trials evaluate investigational adeno-associated virus …