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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
Indication Hemophilia B
Compound Name BE-101 (Padua variant)
Sponsor Be Biopharma
Funder Type Industry
Status
Recruiting
Enrollment Count 24

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 2027-05
Last Update 2024-10-29

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Eligible Sex MALE

Locations


No.of Trial Sites 2
Locations United States

Regulatory Information


Has US IND True
Recent Updates

Resources/Links