Historically, the standard of care for hemophilia A has been intravenous administration of
exogenous factor VIII (FVIII), either as prophylaxis or episodically. The development of …

Adeno-associated virus gene therapy has been the subject of intensive investigation for
monogenic disease gene addition therapy for more than 25 years, yet few therapies have …

Hepatic adeno-associated viral (AAV) gene transfer has the potential to cure the X-linked
bleeding disorder hemophilia A. However, declining therapeutic coagulation factor VIII …

Purpose The anticipated emergence of hemophilia gene therapy will present people with
hemophilia (PWH) and treating clinicians with increasingly complex treatment options. It will …

Introduction Exploring patient perceptions regarding gene therapies may provide insights
about their acceptability to patients. Objective To investigate opinions of people with …

Remarkably, it has been 40 years since the isolation of the two genes involved in
hemophilia A (HA) and hemophilia B (HB), encoding clotting factors VIII (FVIII) and IX (FIX) …

Adeno-associated virus-based gene therapy points to a coming transformation in the
treatment of people living with haemophilia, promising sustained bleed control and potential …

The US Food and Drug Administration (FDA) held a 2-day meeting (September 2–3, 2021)
of the Cellular, Tissue and Gene Therapies Advisory Committee to consider toxicity risks of …

Introduction Haemophilia therapy has evolved from rudimentary transfusion‐based
approaches to an unprecedented level of innovation with glimmers of functional cure …

Hemophilia A gene therapy targets hepatocytes to express B domain deleted (BDD) clotting
factor VIII (FVIII) to permit viral encapsidation. Since BDD is prone to misfolding in the …