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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
NCTID
NCT05456880
(View at clinicaltrials.gov)
Description
This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD
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Indication
Sickle Cell Disease
Compound Name
BEAM-101
Sponsor
Beam Therapeutics Inc.
Funder Type
Industry
Status
Recruiting
Enrollment Count
15
Therapy Information
Target Gene/Variant
HBG1/HBG2
Therapy Type
Gene editing
Therapy Route
Ex-vivo
Mechanism of Action
Mutation correction
Route of Administration
Intravenous
Drug Product Type
Autologous cells
Target Tissue/Cell
CD34+ cells
Delivery System
Electroporation
Vector Type
none
Editor Type
ABE8
Dose 1
Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2022-06-23
Completion Date
2027-02-01
Last Update
2025-01-10
Participation Criteria
Eligible Age
18 Years - 35 Years
Standard Ages
Adult
Eligible Sex
ALL
Locations
No.of Trial Sites
20
Locations
United States
Regulatory Information
Has US IND
True
Recent Updates
Beam expects to dose 30 patients by mid-2025, data update expected mid-2025; 1 patient death reported November 2024, deemed related to busulfan conditioning
Resources/Links
Clinical Publications
(Poster) Impact of BEAM-101 Treatment on Red Blood Cell Hemoglobin Expression, Rheology and Sickling Properties: Initial Data from the BEACON Phase 1/2 Study of Autologous CD34+ Base Edited Hematopoietic Stem Cells in Sickle Cell Disease - ASH 2024
News and Press Releases
Beam Therapeutics Announces Progress in Hematology and Genetic Disease Franchises and Outlines Key 2025 Anticipated Catalysts
Preclinical Publications
(Presentation) Applied Base Editing to Treat Beta Hemoglobinopathies - ASH 2021
(Presentation) Non-genotoxic antibody-based conditioning paired with multi-plex base edited HSCs for the potential treatment of sickle cell disease - FASEB 2022
(Poster) Robust autologous CD34+ HSPC manufacturing with a closed and automated process optimized for patients with sickle cell disease - EHA 2024