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Clinical Trial Report
Gene Therapy Trial Report
Summary
BeCoMe-9: a Clinical Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B
NCTID
NCT06611436
(View at clinicaltrials.gov)
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.
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Development Status
Active
Indication
Hemophilia B
Disease Ontology Term
DOID:12259
Compound Name
BE-101
Compound Description
Padua variant
Sponsor
Be Biopharma
Funder Type
Industry
Recruitment Status
Recruiting
(Click here for study contact information)
Enrollment Count
24
Results Posted
Not Available
Therapy Information
Target Gene/Variant
F9
Therapy Type
Gene editing
Therapy Route
Ex-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Autologous cells
Target Tissue/Cell
B cells
Delivery System
Viral transduction
Vector Type
AAV6
Editor Type
Cas9 RNP
Dose 1
Undisclosed dose 1
Dose 2
Undisclosed dose 2
Dose 3
Undisclosed dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2024-09-18
Completion Date
2027-07
Last Update
2025-03-18
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
3
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Orphan Drug Designation
Recent Updates
Resources/Links
News and Press Releases
FDA Grants Orphan Drug Designation for BE-101, a Novel Engineered B Cell Medicine, for the Treatment of Hemophilia B
Preclinical Publications
(Presentation) Development of an Ex Vivo Precision Gene Engineered B Cell Medicine that Produces Active and Sustained Levels of FIX for the Treatment of Hemophilia B - ASH 2023
Protocol
(Poster) 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 - ASH 2024