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Sirolimus

Beta Thalassemia

Floatz Rating
B60/100
Confidence
Indicative
v0.2
Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
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.
47Moderate confidence
Detailed axis rationale is planned and will be published soon.
TrialPhaseStatusNPrimary endpointReadout
NCT05357482CT.gov
Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia
—Active Not Recruiting———
NCT04247750CT.gov
Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP)
—Unknown———
NCT03877809CT.gov
Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients
—Completed———

Competitive Position

Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
81High confidence
Detailed axis rationale is planned and will be published soon.

Same indication · Beta Thalassemia

AssetSponsorPhaseRating
Sirolimus (this asset)National Heart, Lung, and Blood Institute (NHLBI)P2B · 60
Desferrioxamine BAin Shams UniversityP4BBB
DeferasiroxKevin H.M. Kuo, MD, MSc, FRCPCP4BBB
Autologous Cd34+ Cells Isolated From Mobilised Peripheral Blood By Positive Selection, Modified By Crispr/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats/Crispr-Associated Protein 9) Mediated Gene Editing Consisting Of A Guide Rna (Grna) Introduced Transiently As Ribonucleoprotein (Rnp) Complex, Targeting The Erythroid Lineage-Specific Enhancer Region Of Bcl11A (B-Cell Lymphoma/Leukemia 11A). The Site-Specific Cleavage By Cas9 Forms A Double Strand Break (Dsb), Which Is Subsequently Repaired By Nonhomologous End-Joining (Nhej), Leading To The Transcriptional Repression Of Bcl11A, A Repressor Of ?-Globin Gene TranscriptionDaniel BauerP3BB
HydroxyureaNational Heart, Lung, and Blood Institute (NHLBI)P2/3BB
DeferiproneCarelon ResearchP4BB
ThalidomideNational Institute of Blood and Marrow Transplant (NIBMT), PakistanP2/3BB
LuspaterceptBristol-Myers SquibbP3BB
FludarabineMemorial Sloan Kettering Cancer CenterP2BB

+42 more in the Beta Thalassemia cohort

Other indications for Sirolimus

IndicationSponsorPhaseRating
Tuberous Sclerosis—P3BBB · 73
Disease Related To Transplantation—P4BBB · 72
Myelodysplastic/Myeloproliferative Neoplasm—P2BB · 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.

Citation

Floatz Terminal. Sirolimus in Beta Thalassemia. Methodology v0.2.
Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026.
Accessed July 13, 2026.
https://terminal.floatz.ai/assets/sirolimus-beta-thalassemia

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