CIRM Funded Clinical Trials
A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia
Details:
Sangamo, Inc. is testing genetically engineered blood stem cells for the treatment of beta-thalassemia, a severe form of anemia caused by mutations in the hemoglobin gene. This genetic disorder requires life-long blood transfusions and carries a life expectancy of only 30-50 years. The Sangamo therapy takes a patient’s own blood stem cells and, using a gene-editing technology called zinc finger nuclease (ZFN), provides a functional copy of the hemoglobin gene. These modified cells are given back to the patient which potentially will eliminate the need for chronic transfusions and its associated complications.
Design:
Open label, single arm study
Goal:
Safety and tolerability. Efficacy, change from baseline HbF levels, frequency and volume of RBC transfusions.
