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Platelet Targeted Gene Therapy for Hemophilia A with Inhibitor History

Platelet Targeted Gene Therapy for Hemophilia A with Inhibitor History

January 23, 2025

We report feasibility and safety results after 24 months of follow up for the first participant in a first-in-human trial of gene therapy for severe hemophilia A with factor VIII inhibitor history. Autologous CD34+ cells were transduced with a lentiviral vector encoding factor VIII driven by an ITGA2B gene promoter. This allows platelet-specific expression and storage of the encoded protein in platelet alpha-granules. Factor VIII is released from activated platelets at the injury site to restore hemostasis. Proof-of-concept was demonstrated by vector copy numbers at 6, 12, and 24-months of 0.25, 0.27 and 0.28 copies/cell, in blood, and without spontaneous bleeding or need for exogenous factor VIII.

Tagged: Hematology & Oncology Research – Clinical

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