Gene-Edited cells aim to stop bleeding in severe hemophilia b

NCT ID NCT06611436

Terminated Disease control Sponsor: Be Biopharma Source: ClinicalTrials.gov ↗

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.

hemophilia B moderately severe hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEMOPHILIA B HEMOPHILIA B, MODERATELY SEVERE OR SEVERE

Contacts and locations

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Locations

Georgetown University

Washington D.C., District of Columbia, 20057, United States
University of California, Davis

Davis, California, 95616, United States
University of Michigan

Ann Arbor, Michigan, 48109, United States
University of Minnesota

Minneapolis, Minnesota, 55455, United States
Washington Center for Bleeding Disorders

Seattle, Washington, 98101, United States