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GLOBE1

Development stage: Unknown
Lead developer: Généthon
Modality: Cell Therapies, Gene Therapies
Administration: Intravenous

Overview

GLOBE1 is an autologous ex vivo gene therapy developed by Généthon for the treatment of hemoglobinopathies, including Sickle Cell Disease and beta-thalassemia. The therapy utilizes a lentiviral vector to deliver a functional human beta-globin (HBB) gene into a patient's own CD34+ hematopoietic stem cells (HSCs). These modified cells are then re-infused into the patient, where they are intended to engraft in the bone marrow and produce functional hemoglobin, thereby mitigating the symptoms and complications associated with defective beta-globin production. GLOBE1 is currently being evaluated in Phase 1/2 clinical trials to assess its safety and efficacy in restoring normal hemoglobin levels.

Targets

CD34 (CD34 antigen)

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