Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Enriched Hematopoetic Stem Cell Infusion · 1 trial · 1 indication
Tests are done at key time points to monitor for donor chimerism by evaluating presence of bone marrow-derived hematopoietic stem cells.
| Arm | Type | Description |
|---|---|---|
| Sickle Cell Disease | EXPERIMENTAL | Recipients treated with an enriched hematopoetic stem cell infusion |
| Non-Malignant Disorders | EXPERIMENTAL | Recipients treated with an enriched hematopoetic stem cell infusion |
| Aplastic Anemia | EXPERIMENTAL | Recipients treated with an enriched hematopoetic stem cell infusion |
| Sickle Cell Disease : Extended Protocol | EXPERIMENTAL | Recipients treated with an enriched hematopoetic stem cell infusion and Campath 1H conditioning |
| Name | Type | Description |
|---|---|---|
| Enriched Hematopoetic Stem Cell Infusion | BIOLOGICAL | Enriched Hematopoetic Stem Cell Infusion |
Inclusion Criteria: The following criteria are established to identify subjects with sickle cell disease (SCD) who have a high predicted morbidity and are at risk for early mortality. Subjects with S/S disease, S/C disease, Hemoglobin H disease, Alpha Thalassemia Major, Thalassemia Major (also know...
Top 12 of 13 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 6 | PHASE3 | Etavopivat |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab, OTQ923, ITU512 |
| Agios Pharmaceuticals, Inc. | AGIO | 3 | PHASE3 | Mitapivat, Tebapivat |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, rilzabrutinib |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Travere Therapeutics, Inc. | TVTX | 1 | PHASE3 | L-Citrulline |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470 |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
Enriched Hematopoetic Stem Cell Infusion is an investigational cell therapy being developed by Tourmaline Bio, Inc. for sickle cell disease. It involves infusing enriched hematopoietic stem cells as a treatment approach in hematology. The therapy is in Phase 1 clinical development and has been studied in a single completed trial.
Enriched Hematopoetic Stem Cell Infusion targets ADA, which stands for adenosine deaminase. The target class is an exogenous gene, meaning the therapy introduces a gene to produce the ADA enzyme. This approach is designed to address the underlying defect in sickle cell disease through gene-modified stem cells.
Enriched Hematopoetic Stem Cell Infusion is being developed by Tourmaline Bio, Inc., which trades under the ticker symbol TRML. The company is responsible for the clinical development of this cell therapy for sickle cell disease.
Enriched Hematopoetic Stem Cell Infusion is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA. The only recorded trial is a completed Phase I/II pilot study, and no active trials are currently listed.
Enriched Hematopoetic Stem Cell Infusion has been studied in one completed clinical trial, NCT00777231, titled Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies. The trial enrolled 9 participants with sickle cell disease in the United States. It was a controlled study without randomization or double-blinding.