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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
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 2024-06-26

Participation Criteria

Eligible Age 18 Years - 35 Years
Standard Ages Adult
Eligible Sex ALL

Locations

No.of Trial Sites 15
Locations United States

Regulatory Information

Has US IND True
Recent Updates

Resources/Links

Patents
  • US20200399626A1
  • US11142760B2
  • US20230080198A1
News and Press Releases
Preclinical Publications