By Fineline Cube Shanghai-based BRL Medicine Inc. has announced that it has received clinical trial approval from the Center for Drug Evaluation (CDE) for its BRL-101. The therapy is an autologous hematopoietic stem progenitor cell treatment for transfusion-dependent beta-thalassemia.
Drug Details
BRL-101 is a gene therapy developed through BRL’s in-house developed hematopoietic stem cell platform, ModiHSC. It is primarily designed to treat beta-thalassemia, an inherited hemolytic disorder and one of the most common monogenic disorders. Due to the severe deficiency of functional β-globin, a significant number of patients require regular blood transfusions to survive.
Treatment Mechanism
The platform and product utilize the gene editing system to genetically modify the patient’s hematopoietic stem cells. These modified cells are then re-infused into the patient’s body. Through self-renewal and differentiation, the modified cell population is reconstructed, thereby treating blood system diseases.-Fineline Info & Tech