BRL Medicine Inc., a gene therapy and cellular drug development company, announced on July 22, 2020 that the "Clinical study on the safety and efficacy of autologous hematopoietic stem cell transplantation via gamma-bead protein reactivation in the treatment of beta-thalassemia major", a collaborative study conducted by Bioraylab and Xiangya Hospital Central South University, has achieved preliminary results. The clinical trial achieved initial results. It is worth celebrating that this is the first gene editing treatment for thalassemia in Asia and the first successful treatment of β0/β0 thalassemia major by CRISPR gene editing technology in the world.
The patient's family and the principal investigator of the clinical study, Professor Bin Fu's team from the Department of Hematology, Xiangya Hospital
In this clinical study, BRL Medicine has performed gene editing at specific genomic loci in HSC through CRISPR/Cas9 gene editing technology to activate the patient's own HBG expression, thus dramatically increasing the level of gamma bead protein in red blood cells and bringing the hemoglobin level to the normal range, making it a treatment that may alleviate or even cure the thalassemia.