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A novel thrombin-cleavable FVIII-mimetic antibody enables dynamic, on-demand control of coagulation, reducing adverse thrombotic risk while maintaining efficacy in hemophilia A treatment.
Two hemophilia A patients developed persistent high-titer FVIII inhibitors following AAV-mediated gene therapy with a bioengineered FVIII transgene despite prior extensive FVIII exposure.
This study demonstrates that one-stage assay (OSA) factor VIII activity better reflects the functional enhancement of AAV-derived FVIII in hemophilia A gene therapy, offering superior prediction of bleeding phenotype compared to chromogenic
This study identifies neutrophil-dependent type-I interferon responses and NETs as key drivers of joint damage in hemophilia A, opening avenues for novel therapies to prevent hemophilic arthropathy.
