Innovations in Haemostasis Management in China: From Gene Therapy to Novel Rebalancing Strategies

Haemophilia, a group of inherited bleeding disorders, remains a health challenge due to limitations in traditional factor replacement therapy, including frequent infusions and inhibitor development. Asia, particularly China, has emerged as a hub for innovative haemostasis management, with breakthroughs in gene therapy, novel protein activators and targeted biologics. This paper synthesises key advancements: (1) Gene therapy for haemophilia in China; (2) AAV vector engineering and large-scale manufacturing for haemophilia gene therapy, highlighted by China's first-in-Asia clinical trials and global case reports; (3) the FX activator Bemiltenase Alfa (STSP-0601) for inhibitor-positive patients and (4) the anti-APC monoclonal antibody SR604, a precision therapy under phase I/II trials.