Floatz Rating
BB66/100
Confidence
Indicative
Sponsor
St. Jude Children's Research Hospital
Modality
Small molecule
Development Phase
Phase 2/3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
67High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT07616154CT.gov Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease | — | Not Yet Recruiting | — | — | — |
NCT07373639CT.gov A Long-term Follow-up Study in Patients Who Received BEAM-101 | — | Recruiting | — | — | — |
NCT04774536CT.gov Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease | — | Recruiting | — | — | — |
NCT06506461CT.gov Gene Editing For Sickle Cell Disease | — | Recruiting | — | — | — |
NCT06872333CT.gov Allo HSCT for High Risk Hemoglobinopathies | — | Recruiting | — | — | — |
NCT06399107CT.gov Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease | — | Recruiting | — | — | — |
NCT05456880CT.gov BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease | — | Active Not Recruiting | — | — | — |
NCT04776850CT.gov Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies | — | Withdrawn | — | — | — |
NCT02757885CT.gov Transplantation for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow | — | Completed | — | — | — |
NCT03653247CT.gov A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease | — | Completed | — | — | — |
Show 17 more trialsShow fewer
NCT03653338CT.gov T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias | — | Recruiting | — | — | — |
NCT03263559CT.gov Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507) | — | Completed | — | — | — |
NCT03609840CT.gov Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | — | Completed | — | — | — |
NCT02766465CT.gov Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503) | — | Completed | — | — | — |
NCT02867800CT.gov Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease | — | Completed | — | — | — |
NCT02179359CT.gov Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | — | Terminated | — | — | — |
NCT01565616CT.gov Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease | — | Completed | — | — | — |
NCT01461837CT.gov Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease | — | Active Not Recruiting | — | — | — |
NCT01049854CT.gov CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant | — | Completed | — | — | — |
NCT01279616CT.gov A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease | — | Terminated | — | — | — |
NCT00744692CT.gov Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders | — | Completed | — | — | — |
NCT00578344CT.gov Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP | — | Terminated | — | — | — |
NCT00152113CT.gov Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound | — | Completed | — | — | — |
NCT00176852CT.gov Stem Cell Transplant for Hemoglobinopathy | — | Completed | — | — | — |
NCT00040469CT.gov Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies | — | Terminated | — | — | — |
NCT00029380CT.gov Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia | — | Completed | — | — | — |
NCT00186810CT.gov Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
65High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Sickle Cell Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Busulfanum (this asset) | St. Jude Children's Research Hospital | P2/3 | BB · 66 |
| Glutamine | Ain Shams University | P4 | BBB |
| Hydroxyurea | St. Jude Children's Research Hospital | P4 | BB |
| Crizanlizumab | Novartis Pharmaceuticals | P4 | BB |
| Magnesium Metallicum | Medical College of Wisconsin | P3 | BB |
| Nivestim | St. Jude Children's Research Hospital | P4 | BB |
| Nepenthe | Oman Medical Speciality Board | P4 | BB |
| Fentanyl | University College Dublin | P4 | BB |
| ARG | Tanta University | P3 | BB |
+42 more in the Sickle Cell Disease cohort
Other indications for Busulfanum
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Myelodysplastic/Myeloproliferative Neoplasm | — | P2/3 | BBB · 76 |
| Graft Versus Host Disease | — | P4 | BBB · 74 |
| Disease Related To Hematopoietic Stem Cell Transplant | — | P3 | BBB · 74 |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Busulfanum in Sickle Cell Disease. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/busulfanum-sickle-cell-disease
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