Gene-Edited cells aim to stop bleeding in severe hemophilia b
NCT ID NCT06611436
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
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 episodes. The study will enroll 24 participants to check safety and how well the treatment works.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Georgetown University
Washington D.C., District of Columbia, 20057, United States
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University of California, Davis
Davis, California, 95616, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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Washington Center for Bleeding Disorders
Seattle, Washington, 98101, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A Once-a-Week shot could transform hemophilia Care—Even for those with inhibitors
- Gene Editing's lasting impact: a 10-Year safety watch
- Can a single gene shot free hemophilia b patients from regular infusions?
- Can a video call replace the clinic for hemophilia pain relief?
- Newborn screening study aims to catch rare diseases at birth
- Monthly shot could free kids with hemophilia from frequent infusions