A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants

Summary

This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.

Timeline

Start

2021-07-10

Primary completion

2024-08-23

Completion

2024-12-31

Publications

• Li S, Ling S, Wang D, Wang X, Hao F, Yin L, Yuan Z, Liu L, Zhang L, Li Y, Chen Y, Luo L, Dai Y, Zhang L, Chen L, Deng D, Tang W, Zhang S, Wang S, Cai Y. Modified lentiviral globin gene therapy for pediatric beta0/beta0 transfusion-dependent beta-thalassemia: A single-center, single-arm pilot trial. Cell Stem Cell. 2024 Jul 5;31(7):961-973.e8. doi: 10.1016/j.stem.2024.04.021. Epub 2024 May 16.

Drugs

Indications