Hemophilia
BE-101 is a single IV infusion which does not require preconditioning or immunosuppression in recipients.
Concizumab-mtci improves thrombin production by blocking the tissue factor pathway inhibitor protein.
In the phase 3 explorer8 trial, concizumab reduced the bleeding rate in patients with hemophilia A and B.
Annette Von Drygalski, MD, PharmD, discussed the promising role of etranacogene dezaparvovec gene therapy in hemophilia B.
In a cohort of adults and adolescents marstacimab produced bleed control superior to that from on-demand bypassing agent.
Dr. John Gansner and Dr. Martina H. Slingsby argue this new agent can provide benefit across all bleeding disorders.
Heme Today spoke with Ulrike Reiss, MD, and Andrew Davidoff, MD, of the trial's investigator team.
A preclinical mouse study showed hemostasis restoration, as well as use for active bleeding and internal bleeding prevention.
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