Adeno-associated viruses (AAVs) are the safest and most effective gene delivery vehicles to
drive long-term transgene expression in gene therapy. While animal studies have shown …

Several viral vector-based gene therapy drugs have now received marketing approval. A
much larger number of additional viral vectors are in various stages of clinical trials for the …

Background Concizumab is an anti–tissue factor pathway inhibitor monoclonal antibody
designed to achieve hemostasis in all hemophilia types, with subcutaneous administration …

Regulatory T cells are critical for maintaining immune tolerance. Recent studies have
confirmed their therapeutic suppressive potential to modulate immune responses in organ …

Gene therapy has entered a new era after decades-long efforts, where the recombinant
adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene …

Adeno-associated virus (AAV)-mediated gene transfer has benefited patients with inherited
diseases, such as hemophilia B, by achieving long-term expression of the therapeutic …

Despite current therapies, there remains an unmet need for treatment for patients with
hemophilia. The main parts of two phase 2 trials established clinical proof-of-concept for …

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 …

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 …

Monitoring factor VIII (FVIII) activity has traditionally been complicated by discrepancies
between assays for the various sorts of FVIII molecules. The advent of novel nonfactor …