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BRL-103

Development stage: Phase 2
Lead developer: BRL Medicine
Modality: Gene Silencing → Gene Therapies, Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies
Administration: Ex Vivo (cells Collected From Patient, Edited, Then Reinfused Intravenously)

Overview

BRL-103 is an **autologous gene therapy** involving ex vivo base editing of hematopoietic stem cells (HSCs) from patients with **β-thalassemia major**. The strategy is to reactivate fetal hemoglobin (HbF) production by editing the *BCL11A* enhancer using a glycosylase base editor. This disrupts expression of BCL11A, a key transcriptional repressor of γ-globin genes (*HBG1/2*), thus relieving repression and enabling endogenous fetal hemoglobin production. The edited stem cells are then reintroduced into the patient following conditioning. This approach aims to mitigate the β-globin deficiency seen in β-thalassemia major, potentially reducing or eliminating dependence on chronic transfusions. BRL-103 is developed by Bioray Laboratories (BRL Medicine Inc) and has been tested in clinical trials in China[1][3][4][5].

Other names: γ-globin reactivated autologous hematopoietic stem cells

Targets

BCL11A (B-cell CLL/lymphoma 11A)

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