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. (Show More)
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
Enrollment Count	24
Results Posted	Not Available

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

No.of Trial Sites	3
Locations	United States