On August 16, 2022, BRL Medicine Inc. (hereinafter referred to as "BRL Medicine") announced that its gene therapy product "BRL-101 autologous hematopoietic stem and progenitor cells injection" for transfusion-dependent β-thalassemia ( Pipeline code: BRL-101 ) had obtained Investigational New Drug Application(IND) approval from CDE,NMPA!

Public information on CDE official website

BRL-101: One infusion, lifetime effective

BRL-101 is a gene therapy product developed based on the hematopoietic stem cell platform (ModiHSC®) independently developed by BRL Medicine，of which the main indication is transfusion-dependent β - thalassemia.

β-Thalassemia is an inherited hemolytic disorder that is prevalent worldwide and is one of the most common monogenic disorders. Due to severe deficiency of functional β-globin, a significant proportion of patients require regular blood transfusions to survive, resulting in transfusion-dependent thalassemia (TDT). Due to limited blood resources and high cost of iron chelation agents, only a proportion of domestic TDT patients can maintain standardized blood transfusion and standardized iron removal therapy, and the survival status is worrying. The survival rate of TDT patients is significantly lower than that in developed countries.

BRL Medicine-ModiHSC® mainly uses the gene editing system to genetically modify the patient's hematopoietic stem cells, and the modified hematopoietic stem cells are returned to the patient, and the modified cell population is rebuilt through self-renewal and differentiation, so as to achieve the purpose of treating blood system diseases. At present,BRL Medicine has achieved good results in the IIT clinical research on the treatment of β0/β0 severe thalassemia patients using the self-developed hematopoietic stem cell platform (ModiHSC®) based on gene editing technology.  A quantity of patients with β - thalassemia were freed from transfusion dependence.

Since July 2020,BRL Medicine has cooperated with Xiangya Hospital of Central South University and the No. 923 Hospital of the Joint Logistics Support Force of the Chinese People's Liberation Army. Through BRL-101 gene therapy, many patients with β-thalassemia have been successfully cured and have living a healthy life without dependence on blood transfusion. Among them, 2 patients in Xiangya Hospital of Central South University have been free of blood transfusion dependence for more than 2 years. This is the first time in Asia that gene editing technology has been used to treat thalassemia, and it is also the first successful case in the world that CRISPR gene editing technology has been used to treat β0/β0 severe thalassemia. On August 4, 2022, the results of the clinical trial initiated by the researcher were published in the top international medical academic journal Nature Medicine (impact factor: 87.241), and also represents a breakthrough in clinical research of gene editing therapy in China .

Compared with other β-thalassemia gene therapies costing tens of millions, BRL Medicine-hematopoietic stem cell gene therapy is more convenient, and has the advantages of good targeting, high safety, wide range of action, and significant therapeutic effect. It can achieve a life-long cure with one treatment; and the cost can be greatly reduced, and it is expected to become a therapy that will benefit the public more.