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Two research results of BRL Medcine will be presented at the 65th American Society of Hematology Annual Meeting

2023-11-21

On November 21, 2023, BRL Medcine announced that two studies of BRL-101, a gene therapy product for transfusion-dependent β -thalassemia, and BRL-201, a product for relapsed / refractory B cell non-Hodgkin's lymphoma, were selected for the 65th American Society of Hematology (ASH) Annual Meeting, and the latest data will be announced in the form of poster presentations.


About the BRL-101

1、Research name

Efficacy and Safety of Brl-101, CRISPR-Cas9-Mediated Gene Editing of the BCL11A Enhancer in Transfusion-Dependent β-Thalassemia

2. Abstract number

4995



On August 16, 2022, the clinical trial application (IND) of BRL-101 of BRL Medcine was officially approved by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration and entered the registered clinical stage. Its main indication is transfusion-dependent β-thalassemia, which is a gene therapy product developed based on the hematopoietic stem cell platform (ModiHSC ® ) independently developed by BRL Medcine . ModiHSC® mainly utilizes the gene editing system to genetically modify the hematopoietic stem cells of the patient, and the modified hematopoietic stem cells are infused back into the patient's body to rebuild the modified cell population through self-renewal and differentiation, so as to achieve the purpose of treating hematological diseases . At present, BRL Medcine utilizes the gene therapy of BRL-101 to successfully help many patients with thalassemia to get rid of dependence worldwide.


The conference announced the results of the IIT and IND Phase 1 clinical studies of BRL-101, which is a study conducted in China to evaluate "Safety and Efficacy of γ-globin  reactivated autologous hematopoietic stem cells transplantation for the treatment of severeβ-thalassemia ( thalassemia) ". Thalassemia is an inherited hemolytic disease that is prevalent worldwide and is the largest single-gene mutation genetic disease. Thalassemia patients are mainly due to deletions or mutations of a fragment of the HBB gene, resulting in the severe deficiency of functional β-globin, causing severe anemia and related complications. Whereas hereditary  sustained expression of fetal hemoglobin (HbF) can alleviate the symptoms of anemia, therefore, this clinical study used CRISPR/Cas9-mediated mutation of the BCL11A erythrocyte enhancer to reduce the expression of BCL11A, which can induce the expression of fetal γ-hemoglobin, which is a feasible therapeutic strategy for the treatment of transfusion-dependent β-thalassemia (TDT).


A total of 10 patients aged 6-26 years old were enrolled in the study, and all of them were cured. Clinical treatment results show that the overall Hb and HbF levels increased significantly in all patients undergoing gene-edited HSC transplantation, andthe vast majority of adverse events coincided with hematopoietic stem cell mobilization/apheresis, busulfan myeloablative conditioning and autologous hematopoietic stem cell transplantation throughout the treatment process. All adverse events can be recovered after medical intervention, and the vast majority of adverse events were converted to recovery / resolution. There was no occurrence of GVHD or subject withdrawal from the study or death due to adverse events. In addition, autologous hematopoietic stem progenitor cells (HSPCs) edited by CRISPR/Cas9 were transplanted and differentiated into multiple gene-edited cell lines. As of July 20, 2023, 10 patients were followed up for 24.6 months (4.3-39.2)  after administration  of HSPCs , and 10 patients (100%) were divorced from transfusion dependence, and the longest TI duration has reached 37.2 months. This study shows that 's BRL-101 of BRL Medcine gene therapy is more efficient, convenient and safe, with the advantages of good targeting, high safety, wide range of action ,significant therapeutic effect, etc. It can achieve lifelong cure with one treatment, and is expected to become a more beneficial therapy for the public.


About BRL-201

1. Research name

Long Term Follow-up Results of Brl-201 Phase I Study, a Crispr-Based Non-Viral PD-1 Locus Specific Integrated Anti-CD19 CAR-T Cells in Treating Relapsed or Refractory Non-Hodgkin's Lymphoma

2. Abstract number

2108