BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (BEACON)

A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients With Sickle Cell Disease and Severe Vaso-Occlusive Crises (Beacon Trial)

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

Study Overview

• Status: Active, not recruiting
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Biological: BEAM-101

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • University of Alabama at Birmingham
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Phoenix Children's Hospital
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic Florida
    • Miami, Florida, United States, 33136-1005
      • University of Miami
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Boston Children's Hospital
  • Michigan
    • Detroit, Michigan, United States, 48202
      • Henry Ford Cancer Center
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine in St. Louis
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Hackensack University Medical Center
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
    • New York, New York, United States, 10032
      • Columbia University Irving Medical Center
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Cleveland Medical Center
    • Cleveland, Ohio, United States, 44195
      • The Cleveland Clinic Foundation
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • St Jude Children's Research Hospital
    • Nashville, Tennessee, United States, 37203
      • The Children's Hospital at TriStar Centennial
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Medical College of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 35 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria Include:

1. Age ≥12 years to ≤35 years
2. Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
3. 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:

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BEAM-101; BEAM-101 manufactured with autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and edited ex vivo. No maximum dose has been set for BEAM-101; all of the gene edited cells that pass release specifications will be administered to the patient. BEAM 101 will be administered as a single dose by IV infusion.	Biological: BEAM-101; Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline	6 months to time of analysis as compared to baseline
Proportion of patients with successful neutrophil engraftment	BEAM-101 administration to month 24
Time to neutrophil engraftment	BEAM-101 administration to month 24
Time to platelet engraftment	BEAM-101 administration to month 24
Transplant-related mortality within 100 days after beam-101 treatment	BEAM-101 administration to day 100
Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)	BEAM-101 administration through month 24

Secondary Outcome Measures

Outcome Measure	Time Frame
Proportion of patients experiencing at least 75% reduction in annualized rate of severe VOCs	Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Proportion of patients experiencing no severe VOCs	6 months to time of analysis as compared to baseline
Change in annualized number of hospitalizations for VOCs	Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Change in annualized duration of hospitalizations for VOCs	Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Change in RBC transfusions per month and per year for SCD-related indications	Month 2 post BEAM-101 treatment to month 24 as compared to baseline
Change in total Hgb (g/dL) concentration over time	Baseline to month 24
Proportion of patients with HbF ≥30%, for at least 3 months	Month 6 post BEAM-101 treatment to month 24 as compared to baseline
Change in lactate dehydrogenase (LDH) over time	Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Change in total bilirubin over time	Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Change in free Hgb over time	Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Change in haptoglobin over time	Month 3 post BEAM-101 treatment to month 24 as compared to baseline
Change in reticulocyte count over time	Month 3 post BEAM-101 treatment to month 24 as compared to baseline

Collaborators and Investigators

• Sponsor: Beam Therapeutics Inc.

Publications and helpful links

General Publications

• Ligon JA, Cupit-Link MC, Yu C, Levine J, Foley T, Rotz S, Sharma A, Gomez-Lobo V, Shah NN. Pediatric Cancer Immunotherapy and Potential for Impact on Fertility: A Need for Evidence-Based Guidance. Transplant Cell Ther. 2024 Aug;30(8):737-749. doi: 10.1016/j.jtct.2024.06.006. Epub 2024 Jun 10.
• Sharma A, Young A, Carroll Y, Darji H, Li Y, Mandrell BN, Nelson MN, Owens CL, Irvine M, Caples M, Jerkins LP, Unguru Y, Hankins JS, Johnson LM. Gene therapy in sickle cell disease: Attitudes and informational needs of patients and caregivers. Pediatr Blood Cancer. 2023 Jun;70(6):e30319. doi: 10.1002/pbc.30319. Epub 2023 Mar 28.
• Persaud Y, Mandrell BN, Sharma A, Carroll Y, Irvine M, Olufadi Y, Kang G, Hijano DR, Rai P, Hankins JS, Johnson LM. Attitudes toward COVID-19 vaccine among pediatric patients with sickle cell disease and their caregivers. Pediatr Blood Cancer. 2023 May;70(5):e30274. doi: 10.1002/pbc.30274. Epub 2023 Mar 1.

Study record dates

Study Major Dates

• Study Start (Actual): August 30, 2022
• Primary Completion (Estimated): February 1, 2028
• Study Completion (Estimated): February 1, 2028

Study Registration Dates

• First Submitted: June 23, 2022
• First Submitted That Met QC Criteria: July 11, 2022
• First Posted (Actual): July 13, 2022

Study Record Updates

• Last Update Posted (Estimated): December 16, 2025
• Last Update Submitted That Met QC Criteria: December 15, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Gene Editing; Sickle Cell; Severe Sickle Cell
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Anemia, Sickle Cell
• Other Study ID Numbers: BTX-AUT-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No