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βAS3-FB vector transduced peripheral blood CD34+ cells
- Development stage
- Phase 2
- Lead developer
- UCLA Broad Stem Cell Research Center
- Modality
- Patient-derived iPSCs → iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies, Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Autologous CAR-T → CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Mesenchymal Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Tumor-Infiltrating Lymphocytes (TILs) → Native Immune Cells → Adoptive Cell Transfer → Cell Therapies
- Administration
- Intravenous
01
Overview
A gene therapy product consisting of autologous peripheral blood-derived CD34+ hematopoietic stem and progenitor cells (HSPCs) that have been genetically modified ex vivo using the lentiviral vector βAS3-FB to express an anti-sickling variant of the human β-globin gene (HBB^AS3). The modified HSPCs are reinfused into the patient following myeloablative conditioning. The expressed HBB^AS3 protein inhibits sickle hemoglobin polymerization, thereby reducing red blood cell sickling and ameliorating symptoms of sickle cell disease (SCD). This approach is being developed primarily for SCD and is currently in phase 1/2 clinical trials. The therapy is being developed by researchers at UCLA Broad Stem Cell Research Center in collaboration with the California Institute for Regenerative Medicine[2][4][5].
- Other names
- βAS3-globin gene therapyautologous βAS3-FB vector-transduced CD34+ cells
02
Targets
Hb (Hemoglobin)
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