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Drug intelligence / Profile preview
autologous CD34+ HSPCs electroporated with BCL11A enhancer-targeting Cas9 ribonucleoprotein
- Development stage
- Phase 1
- Lead developer
- Daniel Bauer
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
This investigational autologous cell therapy, developed by Dr. Daniel Bauer at Dana-Farber/Boston Children's Cancer and Blood Disorders Center, utilizes CRISPR-Cas9 technology to treat severe sickle cell disease and transfusion-dependent beta-thalassemia. The process involves collecting a patient's own CD34+ hematopoietic stem and progenitor cells (HSPCs) and using electroporation to deliver Cas9 ribonucleoprotein (RNP) complexes that target and disrupt the erythroid-specific enhancer of the *BCL11A* gene. Since BCL11A normally represses fetal hemoglobin (HbF) production in adults, its lineage-specific knockdown reactivates HbF, which can functionally substitute for defective adult hemoglobin. The edited cells are re-infused into the patient following myeloablative conditioning as a one-time potentially curative treatment.
- Other names
- BCL11A enhancer gene editing therapyBCL-11A enhancer gene editing therapyBCL 11A enhancer gene editing therapyDaniel Bauer BCL11A CRISPR therapyautologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein
02
Targets
BCL11A (B-cell CLL/lymphoma 11A)
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