Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
Status:
Not yet recruiting
Trial end date:
2026-09-10
Target enrollment:
Participant gender:
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)