Floatz Rating
B59/100
Confidence
Indicative
Sponsor
St. Jude Children's Research Hospital
Modality
Small molecule
Development Phase
Phase 2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
55High 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 | — | — | — |
NCT07015684CT.gov 131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD) | — | Recruiting | — | — | — |
NCT06872333CT.gov Allo HSCT for High Risk Hemoglobinopathies | — | Recruiting | — | — | — |
NCT06358638CT.gov Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody | — | Recruiting | — | — | — |
NCT06145282CT.gov Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function | — | Active Not Recruiting | — | — | — |
NCT06078696CT.gov Siplizumab for Sickle Cell Disease Transplant | — | Terminated | — | — | — |
NCT05384756CT.gov TMLI and Alemtuzumab for Treatment of Sickle Cell Disease | — | Active Not Recruiting | — | — | — |
NCT05357482CT.gov Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia | — | Active Not Recruiting | — | — | — |
NCT04362293CT.gov Reduced Intensity Transplantation for Severe Sickle Cell Disease | — | Suspended | — | — | — |
NCT03587272CT.gov Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease | — | Recruiting | — | — | — |
Show 14 more trialsShow fewer
NCT03421756CT.gov Stem Cell Transplant in Patients With Severe Sickle Cell Disease | — | Terminated | — | — | — |
NCT03214354CT.gov Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor | — | Recruiting | — | — | — |
NCT03077542CT.gov Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease | — | Active Not Recruiting | — | — | — |
NCT03121001CT.gov Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease | — | Recruiting | — | — | — |
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 | — | — | — |
NCT02678143CT.gov Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease | — | Terminated | — | — | — |
NCT02105766CT.gov Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure | — | Active Not Recruiting | — | — | — |
NCT02013375CT.gov SCD-Haplo: Phase II Study of HLA-Haploidentical SCT for Aggressive SCD | — | Terminated | — | — | — |
NCT01850108CT.gov Non-Myeloablative Conditioning and Bone Marrow Transplantation | — | Unknown | — | — | — |
NCT01499888CT.gov Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD) | — | Active Not Recruiting | — | — | — |
NCT00977691CT.gov Haploidentical PBMC Transplant for Severe Congenital Anemias | — | Active Not Recruiting | — | — | — |
NCT00489281CT.gov Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders | — | Terminated | — | — | — |
NCT00061568CT.gov Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias | — | Unknown | — | — | — |
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 |
|---|---|---|---|
| Sirolimus (this asset) | St. Jude Children's Research Hospital | P2 | B · 59 |
| Glutamine | Ain Shams University | P4 | BBB |
| Hydroxyurea | St. Jude Children's Research Hospital | P4 | BB |
| Crizanlizumab | Novartis Pharmaceuticals | P4 | BB |
| Nivestim | St. Jude Children's Research Hospital | P4 | BB |
| Magnesium Metallicum | Medical College of Wisconsin | P3 | BB |
| ARG | Tanta University | P3 | BB |
| Fentanyl | University College Dublin | P4 | BB |
| Nepenthe | Oman Medical Speciality Board | P4 | BB |
+42 more in the Sickle Cell Disease cohort
Other indications for Sirolimus
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Tuberous Sclerosis | — | P3 | BBB · 73 |
| Disease Related To Transplantation | — | P4 | BBB · 72 |
| Myelodysplastic/Myeloproliferative Neoplasm | — | P2 | BB · 69 |
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. Sirolimus 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/sirolimus-sickle-cell-disease
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