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 (Show More)
Development Status	Active
Indication	Sickle Cell Disease
Disease Ontology Term	DOID:0081445
Compound Name	BEAM-101
Sponsor	Beam Therapeutics Inc.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	15 (ESTIMATED)
Results Posted	Not Available

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 (≥3.0E6 viable CD34+ cells/kg)
Dose 2	Range: 3.2-23.4E6 CD34+ cells/kg, N=17
Dose 3
Dose 4
Dose 5

No.of Trial Sites	20
Locations	United States

Has US IND	True
FDA Designations	Regenerative Medicine Advanced Therapy
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