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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


Sickle Cell Disease

Recruitment Status


Eligibility Criteria

Key Inclusion Criteria Include:

Age ≥18 years to ≤35 years for the initial sentinel cohort; for subsequent enrollment patients from ≥12 years up to ≤35 years may be enrolled only upon approval by FDA.
Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
Severe SCD defined by the occurrence of at least 4 severe VOCs in the 24 months prior to screening despite receiving hydroxyurea or other supportive care measures

Key Exclusion Criteria Include:

HbF levels >20%, obtained at the time of screening on or off hydroxyurea therapy
Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
Available and willing matched sibling donor
Definitive diagnosis of moyamoya syndrome based on screening brain MRA
History of overt stroke


Intervention Type

Intervention Name




Phase 1, Phase 2



Min Age

18 Years

Max Age

35 Years

Download Date

May 17, 2024

Principal Investigator

Primary Investigator: Matthew Heeney, MD

Co-Investigator: David Williams, MD

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Primary Contact Information

Medical Information


For more information on this trial, visit


For more information and to contact the study team:

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease NCT05456880 Medical Information 857-327-8641