On February 17, 2023, BRL Medicine Inc. (hereinafter referred to as "BRL Medicine") ushered in another good news. Its gene editing treatment product for transfusion-dependent β-thalassemia (pipeline code: BRL -101) multi-center phase I clinical trial, cured the first adult severe thalassemia in the First Affiliated Hospital of Guangxi Medical University. The patient was successfully discharged on January 16, 2023, and has reached the point of getting rid of blood transfusion dependence Standards. This breakthrough is undoubtedly a shot in the arm for domestic and foreign medical circles, which helps patients of β-thalassemia to see the hope of life again.
When discharged from the hospital, the first patient (left 5) took a group photo with Professor Lai Yongrong (PI, left 4) and medical staff
The first patient in this trial was a 21-year-old college student who was diagnosed with β+/β0 thalassemia 8 months after birth. Before receiving gene therapy, the patient had maintained routine blood transfusion and iron removal treatment, the average annual transfusion of whole blood is as high as 15042ml. The patient completed the first drug infusion on December 8, 2022. After the gene-edited HSC transplantation treatment, the patient's hemoglobin began to increase significantly, reaching 94.4g/L on the 37th day, successfully achieving hematopoietic stem cell implantation and Hematopoietic reconstruction. It is worth mentioning that no severe infection or other adverse events occurred during the entire treatment process, and he was successfully discharged from the hospital in only 40 days, and has now reached the standard of getting rid of blood transfusion dependence. Now that he can return to normal life, the patient also said excitedly: "Finally, I can realize my study plan with peace of mind!"
The clinical trial is a multi-center, open-label clinical study designed to evaluate the safety and efficacy of single-dose intravenous infusion of BRL-101 in the treatment of transfusion-dependent β-thalassemia. Previously, BRL Medicine had achieved good results in the Investigator Initiated Trial(IIT). It successfully cured 6 patients with β-thalassemia worldwide, and all of them got rid of blood transfusion dependence for more than one year, of which 2 patients had been free from blood transfusion dependence for more than 2 and a half 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. The results of this early clinical research were published in the top international medical academic journal Nature Medicine on August 4, 2022 .
About Beta -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 in China is significantly lower than that in developed countries. Among the current traditional treatments for thalassemia, hematopoietic stem cell transplantation is the only method that can cure β-thalassemia, but it is expensive and extremely difficult to match. Only a small number of patients can obtain transplantation. If autologous hematopoietic stem cells can be reinfused into the patient after gene correction , the problem of insufficient source of hematopoietic stem cells and difficulty in matching can be solved, and the progress of CRISPR gene editing technology provides the feasibility for this therapeutic strategy.
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.. It had obtained Investigational New Drug Application(IND) approval in August 2022 from CDE,NMPA! In October of the same year, the multi-center phase 1/2 clinical trial of BRL-101 was officially launched. ModiHSC ® mainly uses the gene editing system to genetically modify the patient's hematopoietic stem cells, and the modified hematopoietic stem cells are reinfused into the patient's body, and the modified cell population is rebuilt through self-renewal and differentiation, so as to achieve the purpose of treating blood system diseases. 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.
Based on gene editing technology of the hematopoietic stem cell platform (ModiHSC ® ), BRL Medicine has achieved good results in the clinical trials of the treatment of patients with β0/β0 severe thalassemia, and has successfully helped many patients with β0 -Thalassemia patients get rid of blood transfusion dependence.