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Moderately severe hemophilia B

MONDO:0015716

Moderately severe hemophilia B is a form of hemophilia B characterized by factor IX deficiency leading to abnormal bleeding as a result of minor injuries, or following surgery or tooth extraction.

Also known as: moderately severe factor IX deficiency, moderately severe haemophilia type B, moderately severe hemophilia type B

14 clinical trials for this condition and its sub-types.

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Broader categories

Disease (680) Hereditary disease (176) Hematologic disorder (170) Hemorrhagic disease (103) Hemophilia B (83) Blood coagulation disease (53) Hemophilia (53) Human disease (14) Coagulation protein disease (13) Inherited blood coagulation disorder (8)
Trials to join now! 8 Not yet recruiting 2 Not yet finished but already full! 1 Completed 2 Terminated 1
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  • Gene-Edited cells aim to stop bleeding in severe hemophilia b

    Disease control Terminated

    This early-stage trial tests a one-time treatment called BE-101 for adults with severe hemophilia B. The therapy uses a person's own blood cells, modified with CRISPR gene editing, to continuously produce the missing clotting factor. The goal is to reduce or prevent bleeding epis…

    Phase: PHASE1, PHASE2 • Sponsor: Be Biopharma • Aim: Disease control

    Last updated Jun 27, 2026 14:00 UTC

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