Study Summary
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in subjects with Transfusion-Dependent β-Thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101)
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BRL-101DRUG
CD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A enhancer site
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Nanfang Hospital, Southern Medical University | Guangzhou | Guangdong | China |
| The First Affiliated Hospital of Guangxi Medical University | Nanning | Guangxi | China |
| Xiangya Hospital of Central South University | Changsha | Hunan | China |
| Chinese Academy of Medical Sciences | Tianjin | Tianjin Municipality | China |