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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
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
Enrollment Count 24 (ESTIMATED)
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

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-09-18
Completion Date 2027-07
Last Update 2025-10-20

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria: * Adult Males (≥18) with moderately severe to severe Hemophilia B (FIX deficiency) * Received ≥50 exposure days to Factor IX products preceding enrollment. * Currently receiving prophylaxis treatment * Adequate organ function and clinical labs * Able to tolerate study procedures including leukapheresis. Exclusion Criteria: * Pre-existing or history of specific diseases * B-Cell malignancy, EBV lymphoproliferative disease * Primary immunodeficiency disease or disorder (PIDD) or systemic immuno-suppression * Arterial and/or venous thromboembolic events within 2 years prior to dosing * History of anaphylaxis or nephrotic syndrome * Active infection (HIV, Hep B or C) * History of inhibitor to FIX or inhibitor * History of an allergic reaction or anaphylaxis to FIX products * Planned surgical procedure within 6 months from BE-101 administration * Previously dosed with gene therapy * Participated in an interventional study and/or received an interventional study drug within 30 days or five-half-lives (whichever is longer) of consent into BeCoMe-9 and for the duration of the study * Planned participation in clinical trial within one year after BE-101 * Administration of an investigational agent or vaccine within 28 days of leukapheresis and dosing of BE-101 Other protocol-defined inclusion/exclusion criteria may apply.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates First patient dosed July 2025

Resources/Links