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 …

Haemophilia B is a rare X-linked genetic deficiency of coagulation factor IX (FIX) that, if
untreated, can cause recurrent and disabling bleeding, potentially leading to severe …

Recombinant prophylactic treatment (PTX) has greatly improved morbidity, mortality and
health‐related quality of life (HRQoL) in patients with severe haemophilia. Yet, treatment …

Introduction Valoctocogene roxaparvovec uses an adeno‐associated virus serotype 5
(AAV5) vector to transfer a factor VIII (FVIII) coding sequence to individuals with severe …

Gene transfer therapies utilizing adeno-associated virus (AAV) vectors involve a complex
drug design with multiple components that may impact immunogenicity. Valoctocogene …

Background: Hemophilia A is a chronic condition that requires meticulous treatment and
management. Patient preferences for prophylactic treatment can substantially influence …

Introduction Gene therapy has shown promise in clinical trials for patients with haemophilia,
but patient preference studies have focused on factor replacement treatments. Aim We …

Abstract Background and Objective New treatments for haemophilia are under development
or entering the market, including extended half-life products, designer drugs and gene …

Background The recombinant factor VIII (rFVIII)-IgG1 Fc fusion protein (rFVIII-Fc) was the first
available extended half-life rFVIII, shown to prolong dosing intervals of individualised …

Introduction Head‐to‐head evaluation of valoctocogene roxaparvovec, the first gene therapy
approved for haemophilia A, with emicizumab is not available. Therefore, phase 3 trial data …