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GbG lentiviral gamma-globin vector
- Development stage
- Unknown
- Lead developer
- Aruvant Sciences
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
The GbG lentiviral gamma-globin vector is an ex vivo gene therapy designed for the treatment of sickle cell disease and other hemoglobinopathies. It utilizes a lentiviral vector to deliver the human Aγ-globin gene into autologous hematopoietic stem cells (HSCs). The transgene expression is controlled by β-globin regulatory elements, including the β-globin promoter and locus control region (LCR) hypersensitive sites 2, 3, and 4. By increasing the production of fetal hemoglobin (HbF), the therapy aims to inhibit the polymerization of sickle hemoglobin (HbS), thereby preventing red blood cell sickling and reducing clinical complications. A variant of this vector, GbGM, contains a G16D mutation in the γ-globin gene to further enhance its anti-sickling potency. The development of this therapy has been led by researchers at Cincinnati Children's Hospital Medical Center.
- Other names
- GbG lentiviral vectorgamma-globin lentiviral vector
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