Sirolimus
Severe Combined Immunodeficiency
Floatz Rating
B60/100
Confidence
Indicative
Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
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.
45High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04370795CT.gov Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | — | Withdrawn | — | — | — |
NCT02177760CT.gov Sirolimus Prophylaxis for aGVHD in TME SCID | — | Withdrawn | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Severe Combined Immunodeficiency
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Sirolimus (this asset) | National Institute of Allergy and Infectious Diseases (NIAID) | P2 | B · 60 |
| Fludarabine | National Institute of Allergy and Infectious Diseases (NIAID) | P2/3 | BB |
| Nivestim | National Institute of Allergy and Infectious Diseases (NIAID) | P2 | BB |
| Alemtuzumab | Washington University School of Medicine | P2/3 | BB |
| Busulfanum | National Institute of Allergy and Infectious Diseases (NIAID) | P2/3 | BB |
| Cyclophosphamide | St. Jude Children's Research Hospital | P2/3 | BB |
| Elapegademase | Leadiant Biosciences, Inc. | P3 | BB |
| Thiotepa | St. Jude Children's Research Hospital | P2 | BB |
| Adagen | Leadiant Biosciences, Inc. | P3 | BB |
+22 more in the Severe Combined Immunodeficiency 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.
Citation
Floatz Terminal. Sirolimus in Severe Combined Immunodeficiency. 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-severe-combined-immunodeficiency
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