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BRL-101

Development stage: Phase 2
Lead developer: BRL Medicine
Modality: Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies
Administration: Intravenous

Overview

BRL-101 is an autologous, genetically modified hematopoietic stem cell gene therapy developed by BRL Medicine for the treatment of transfusion-dependent beta-thalassemia (TDT) and sickle cell disease. The therapy uses CRISPR/Cas9 gene editing to disrupt the erythroid-specific enhancer region of the BCL11A gene in CD34+ hematopoietic stem and progenitor cells. This disruption reduces BCL11A expression, leading to increased production of fetal hemoglobin (HbF), which can compensate for defective or absent adult hemoglobin in patients with beta-thalassemia or sickle cell disease. The edited cells are reinfused into the patient after myeloablation, where they engraft and repopulate the blood system with corrected cells. Clinical trials have shown that BRL-101 can induce transfusion independence in TDT patients and increase total hemoglobin and HbF levels[1][3][6]. The product is based on BRL Medicine’s ModiHSC platform.

Other names: Autologous Hematopoietic Stem Cells (BRL Medicine)

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