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Evaluating BEAM-101 for severe sickle cell disease

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)

PHASE1; PHASE2 · Beam Therapeutics Inc. · NCT05456880

This study is testing a new treatment called BEAM-101 to see if it can help young people with severe sickle cell disease who have not found relief from standard treatments.

Quick facts

PhasePHASE1; PHASE2
Study typeInterventional
Enrollment15 (estimated)
Ages18 Years to 35 Years
SexAll
SponsorBeam Therapeutics Inc. (industry)
Locations20 sites (Birmingham, Alabama and 19 other locations)
Trial IDNCT05456880 on ClinicalTrials.gov

What this trial studies

This open-label, single-arm, multicenter Phase 1/2 study assesses the safety and efficacy of BEAM-101, an autologous base edited CD34+ hematopoietic stem and progenitor cells (HSPCs), in patients with severe sickle cell disease (SCD). The study aims to enroll patients aged 12 to 35 years who have a documented diagnosis of severe SCD and have experienced multiple severe vaso-occlusive crises despite standard treatments. Participants will receive BEAM-101 to evaluate its potential to improve their condition.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 12 to 35 years with severe sickle cell disease who have had at least four severe vaso-occlusive crises in the past two years.

Not a fit: Patients with HbF levels greater than 20%, previous stem cell transplants, or those with a matched sibling donor may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the frequency of severe vaso-occlusive crises in patients with severe sickle cell disease.

How similar studies have performed: Other studies involving gene editing for sickle cell disease have shown promise, indicating potential for success with this novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria Include:

1. 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.
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

Where this trial is running

Birmingham, Alabama and 19 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Sickle Cell Disease, Gene Editing, Sickle Cell, Severe Sickle Cell

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.