Clinical Trial

Disease: Hemophilia B, (NCT06611436)

Disease info:

Haemophilia B is a bleeding disorder that slows the blood clotting process. People with this disorder experience prolonged bleeding or oozing following an injury or surgery. In severe cases of hemophilia, heavy bleeding occurs after minor injury or even in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. Milder forms may not become apparent until abnormal bleeding occurs following surgery or a serious injury. Hemophilia B is caused by mutations in the F9 gene which encodes coagulation factor IX.

Hemophilia primarily affects males, but a milder, symptomatic form of hemophilia B in female carriers has also been described.

Frequency:
The incidence of Haemophilia B is roughly 1 case per 25,000-30,000 male births.
Official title:
BeCoMe-9: a Phase 1/2 Dose Escalation and Expansion Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B
Who:

Name: Be Biopharma

Email: askhemophiliab@be.bio

Sponsor:
Partners:
Locations:

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

United States, Minnesota
Minneapolis, Minnesota, United States, 55455
Recruiting
University of Minnesota

United States, Washington
Seattle, Washington, United States, 98101
Recruiting
Washington Center for Bleeding Disorders

Study start:
Dec. 4, 2024
Enrollment:
24
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene insertion
Gene:
FIX (human factor IX)
Delivery method:
Adeno-associated virus (AAV) - Ex-vivo
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

The BeCoMe-9 Study (BE-101-01) is a Phase 1/2, first in human, multi-center, open-label, dose-escalation study to evaluate the safety and clinical activity of a single intravenous (IV) dose of BE-101 in adults with moderately severe or severe Hemophilia B. Once infused, BE-101 is designed to engraft and continuously secrete FIX into the circulation to restore clinically meaningful levels of active FIX. BE-101 is an autologous (person's own cells) B Cell Medicine (BCM) which uses CRISPR/Cas9 gene editing to precisely insert human FIX gene into those cells.

Last updated: Jun. 15, 2025
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