A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia
Grant Award Details
Grant Type:
Grant Number:
CLIN2-11031
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Cell Line Generation:
Award Value:
$5,150,000
Status:
Closed
Grant Application Details
Application Title:
A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia
Public Abstract:
Therapeutic Candidate or Device
ST-400 is a gene-edited cell therapy candidate for patients with transfusion-dependent beta-thalassemia
Indication
Transfusion-dependent beta-thalassemia
Therapeutic Mechanism
ST-400 is intended to disrupt BCL11A erythroid enhancer in CD34+ HSPC resulting in an increase in fetal hemoglobin which can substitute for reduced or absent adult Hb. Therefore, treatment with ST-400 may potentially reduce or eliminate need for chronic blood transfusions and likely improve quality of life of patients
Unmet Medical Need
Beta-thalassemia patients require life-long blood transfusions which result in iron overload in many organs. Consequently, patients require iron chelators to treat iron overload. Overall, thalassemia patients have lower quality of life and shorter lifespan compared to overall US population
Project Objective
Safety established, efficacy/activity observed
Major Proposed Activities
ST-400 is a gene-edited cell therapy candidate for patients with transfusion-dependent beta-thalassemia
Indication
Transfusion-dependent beta-thalassemia
Therapeutic Mechanism
ST-400 is intended to disrupt BCL11A erythroid enhancer in CD34+ HSPC resulting in an increase in fetal hemoglobin which can substitute for reduced or absent adult Hb. Therefore, treatment with ST-400 may potentially reduce or eliminate need for chronic blood transfusions and likely improve quality of life of patients
Unmet Medical Need
Beta-thalassemia patients require life-long blood transfusions which result in iron overload in many organs. Consequently, patients require iron chelators to treat iron overload. Overall, thalassemia patients have lower quality of life and shorter lifespan compared to overall US population
Project Objective
Safety established, efficacy/activity observed
Major Proposed Activities
- Manufacture ST-400 for each subject in the proposed Phase 1 / 2 clinical study
- Assess safety, tolerability, biological activity, and clinical efficacy of ST-400 over a 52-week period
- Decision to continue development; plan Phase 3 study
Statement of Benefit to California:
About 200 beta-thalassemia patients are estimated to live in CA; ST-400 may be a useful for a substantial portion of these patients. Sangamo is an emerging California-based biotechnology company with about 170 employees in Richmond, CA. Two of the proposed clinical sites for the study are California based (Benioff Children's and UCLA). ST-400 will be manufactured by HCATS based in Mountain View, CA. Overall, this project will deliver significant medical and economic benefit to California