Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05456880 | BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease | PHASE1 | ACTIVE NOT_RECRUITING | 15 | — | — | Aug 30, 2022 | Feb 1, 2028 | Dec 16, 2025 | 19 | United States |
| Arm | Type | Description |
|---|---|---|
| BEAM-101 | EXPERIMENTAL | 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. |
| Name | Type | Description |
|---|---|---|
| BEAM-101 | BIOLOGICAL | Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan |
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...