On August 4, 2022, BRL Medicine Inc. (hereinafter referred to as " BRL Medicine ") announced that he world's first CRISPR gene editing treatment of children with β0/β0 severe thalassaemia in cooperation between BRL Medicine and Xiangya Hospital of Central South University, has been free from blood transfusion dependence for more than 2 years and has started its normal life and study. Meanwhile, the results of the clinical trial were published today in Nature Medicine, a top international medical academic journal (Impact Factor: 87.241).
As the first clinical study based on CRISPR gene editing in the treatment of β0/β0 severe thalassemia published in a top academic journal, the article published detailed clinical data and more comprehensively interpreted the efficacy and safety characteristics of patients treated with BRL-101.
Gene therapy, one treatment can achieve a lifelong cure
Inducing the expression of γ-globin (fetal hemoglobin HbF) by gene editing is a highly promising strategy for the treatment of β-thalassemia caused by mutations in the HBB gene. The team of BRL Medicine published the clinical data of the therapy in detail today in an article in Nature Medicine , and evaluated the safety and efficacy of gene therapy in children with transfusion-dependent β-thalassemia (TDT).
The trial transplanted gene-edited autologous hematopoietic stem progenitor cells ( HSPCs) into two pediatric patients, one of whom had a genotype of β0/β0, was classified as the most severe type of TDT; Another treated child also had TDT. As of the time of submission of the article, the fetal hemoglobin of the two children increased from 2.55g/L and 1.75g/L at the baseline to 149g/L and 139g/L at the latest visit ,respectively, and the total hemoglobin content also reached 152g /L and 140g/L, and have achieved freedom from blood transfusion dependence for more than 16 months after treatment (freedom from transfusion dependence is defined as a total hemoglobin of 90 g/L or more without transfusion). The toxicity associated with myeloablative pretreatment was mild throughout the course of treatment , no serious infection occurred, and they were discharged at 52 and 40 days after transplantation, respectively . As shown in the figure below, the number of red blood cells and overall hemoglobin level in both patients began to increase steadily around day 45 and reached healthy levels around day 75. As of now, the two subjects had been free from blood transfusion dependence for more than 24 months.