WebDec 26, 2024 · This is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major. WebApr 28, 2024 · Phase 1. Detailed Description: This phase 1 clinical study is an open-label, multiple-dose, dose-escalation, dose-expansion, safety, PK, PD study of BR105. The phase 1 protocol will have 2 parts: a single agent dose escalation phase (Part 1) and dose expansion phase (Part 2). Approximately 40-162 adult patients are expected to be …
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