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ST-400 Investigational product · 1 trial · 1 indication
Safety and tolerability assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 156 weeks after the ST-400 infusion
| Arm | Type | Description |
|---|---|---|
| ST-400 Investigational product | EXPERIMENTAL | ST-400 Investigational product is composed of autologous CD34+ hematopoietic stem/progenitor cells that are genetically modified ex vivo at the erythroid-specific enhancer of the BCL11A gene |
| Name | Type | Description |
|---|---|---|
| ST-400 Investigational product | GENETIC | Single dose of ST-400 following chemotherapy conditioning with busulfan |
Inclusion Criteria: 1. Informed Consent 2. Clinical diagnosis of TDT with ≥ 8 documented RBC transfusion events per year on an annualized basis in the 2-years prior to screening 3. Confirmed beta-thalassemia diagnosis by molecular genetic testing 4. Clinically stable and eligible to receive conditi...
ST-400 is an investigational gene therapy being studied for the treatment of transfusion-dependent beta-thalassemia (TDT), a severe blood disorder requiring regular blood transfusions. It is designed to address the underlying genetic cause of the condition. ST-400 is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
ST-400 is being developed by Sangamo Therapeutics, Inc., a biotechnology company traded on the Nasdaq under the ticker symbol SGMO. The company is conducting clinical research to evaluate the safety, tolerability, and efficacy of ST-400 in patients with transfusion-dependent beta-thalassemia.
ST-400 is in Phase 1 clinical development. A Phase 1 study of ST-400 has been completed, involving 5 participants with transfusion-dependent beta-thalassemia. The study was uncontrolled, meaning there was no comparison group, and it was not randomized or double-blinded. ST-400 remains investigational and has not been approved by the FDA.
ST-400 has been studied in one clinical trial, identified as NCT03432364, titled "A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)." This Phase 1 trial was conducted in the United States and enrolled 5 adult participants aged 18 years and older. The trial has been completed.
No, ST-400 is not FDA approved. It is an investigational gene therapy currently in Phase 1 clinical development for transfusion-dependent beta-thalassemia. The completed Phase 1 trial assessed safety, tolerability, and efficacy, but ST-400 has not yet received regulatory approval for any use.