Long-Term Efficacy and Safety of BRL-101, CRISPR-Cas9-mediated Gene Editing of The BCL11A Enhancer for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 5.6 Years of Follow-up

busulfan - Generic mfg.

BRL-101 - BRL Medicine

https://pag.virtual-meeting.org/US/asgct2026/en-US/pag/presentation/686670

Rongrong Liu1; Qian Tan2; Xinhua Zhang3; Li Wang3; Bin Fu4; Yajing Xu4; Jun Shi5; Xiaoqin Feng6; Sixi Liu7; Rongmei Liang2; Lingzhi Tan2; Xiaoxia MA2; Yu Xiang2; Dali Li2; Yuxuan Wu2; Mingyao Liu2; Jianpei Fang8; Yongrong Lai1

Mar 22, 2026