Recombinant adeno-associated virus (rAAV) gene therapy has the potential to transform the
lives of patients with certain genetic disorders by increasing or restoring function to affected …

Adeno-associated viral (AAV) vector gene therapy has shown promise as a possible cure for
hemophilia. However, immune responses directed against AAV vectors remain a hurdle to …

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 …

Gene therapy is at the forefront of the drive to bring the potential of cure to patients with
genetic diseases. Multiple mechanisms of effective and efficient gene therapy delivery (eg …

Haemophilia is an X-linked lifelong congenital bleeding disorder that is caused by
insufficient levels of factor VIII (FVIII; haemophilia A) or factor IX (FIX; haemophilia B) and …

Gene therapy for hemophilia using adeno-associated virus (AAV) derived vectors can
reduce or eliminate patients' disease-related complications and improve their quality of life …

The introduction and development of biologics such as therapeutic proteins, gene-, and cell-
based therapy have revolutionized the scope of treatment for many diseases. However, a …

Introduction Current treatment for haemophilia A involves factor VIII replacement or non‐
replacement (emicizumab) therapies, neither of which permanently normalise factor VIII …

Hemophilia A, a single gene disorder leading to deficient Factor VIII (FVIII), is a suitable
candidate for gene therapy. The aspiration is for single administration of a genetic therapy …

Recurrent joint bleeds lead to progressive arthropathy, which is the main long-term
complication for patients with haemophilia. Prophylactic replacement therapy from early age …