DATE2025.11.07 #Press Releases

Advancing Domestic Gene Therapy for Hemophilia A

-Development of a Modified, Functionally Enhanced Factor VIII-

Summary

Associate Professor Hiroshi Kashiwakura and Professor Tsukasa Omori of the Department of Pathophysiological Biochemistry, Division of Biochemistry, Jichi Medical University School of Medicine; Professor Keiji Nogami of the Department of Pediatrics, Nara Medical University; Professor Osamu Nureki of the Graduate School of Science, The University of Tokyo; Dr. Tiago Lopes of Nezu Life Sciences (now Nezu Biotech GmbH); Professor Susumu Uchiyama from the Graduate School of Engineering, The University of Osaka, and a research group from the Japan Society for the Prevention of Disease have successfully developed a highly functional modified blood coagulation Factor VIII (FVIII). This was achieved by comparing the amino acid sequences of coagulation Factor VIII across animal species, resulting in a significant increase in coagulation factor activity and secretion capacity while reducing endoplasmic reticulum stress. This result suggests the potential for achieving therapy with a significantly lower vector dose compared to existing hemophilia A gene therapies (Roctavian®) using adeno-associated virus (AAV) vectors. If this high-functionality FVIII enables a reduction in the dosage of hemophilia A gene therapy drugs, it could lead to lower treatment costs and reduced risk of side effects, potentially enhancing both practicality and safety.

Figure：Effective gene therapy for hemophilia A using modified FVIII

Links

Jichi Medical University, Nara Medical University, Osaka University

Journals

Journal name	Blood
Title of paper	Engineered coagulation factor VIII with enhanced secretion and coagulation potential for hemophilia A gene therapy