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VGB-Ex01
- Development stage
- Phase 1
- Lead developer
- Shanghai Vitalgen BioPharma
- Modality
- Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Therapies, Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies
- Administration
- Parenteral
01
Overview
VGB-Ex01 is an autologous gene therapy under development for the treatment of beta-thalassemia. The therapy involves ex vivo editing of a patient's own hematopoietic stem cells using the CRISPR-Cas12b system to modify the HBG1/2 promoter region, with the goal of reactivating fetal hemoglobin (HbF) production and reducing or eliminating transfusion dependence. The edited stem cells are then administered back to the patient via parenteral infusion. This approach aims to provide a one-time, potentially curative treatment for transfusion-dependent beta-thalassemia patients. The drug is being developed by Shanghai Vitalgen BioPharma and is currently in Phase II clinical trials[1][2][3][5][7].
- Other names
- CRISPR-Cas12b editing hematopoietic stem cellsCRISPR-Cas-12b editing hematopoietic stem cellsCRISPR-Cas 12b editing hematopoietic stem cells
02
Targets
HBG1/HBG2 (Gamma-globin genes)
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