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transfusion-dependent β-thalassemia

1 Article
Illustration of BRL-101 gene therapy for transfusion-dependent β-thalassemia, showing stem cell collection, CRISPR-Cas9 editing of BCL11A, fetal hemoglobin reactivation and restoration of healthy red blood cells.

BRL Medicine’s BRL-101 Meets Primary Endpoint in Pivotal Trial for Transfusion-Dependent β-Thalassemia

By Henry Tseng
No Comments on BRL Medicine’s BRL-101 Meets Primary Endpoint in Pivotal Trial for Transfusion-Dependent β-Thalassemia
7 Min Read

BRL Medicine’s gene-edited stem cell therapy BRL-101 has met the prespecified primary efficacy endpoint in a pivotal Chinese study for transfusion-dependent β-thalassemia, with all 30 treated patients achieving transfusion independence and the…