SAR445136
- Generic Name
- SAR445136
- Drug Type
- Biotech
Overview
SAR445136 is an investigational zinc finger nuclease gene-edited cell therapy. It consists of autologous CD34+ hematopoietic stem precursor cells (HSPC) modified ex vivo by zinc finger nucleases (ZFN) that target the BCL11A gene erythroid-specific enhancer (ESE) to increase endogenous HbF production in erythrocytes (RBC). It was formerly investigated for the treatment of beta-thalassemia, and it is currently being investigated for the treatment of sickle cell disease.
Background
SAR445136 is an investigational zinc finger nuclease gene-edited cell therapy. It consists of autologous CD34+ hematopoietic stem precursor cells (HSPC) modified ex vivo by zinc finger nucleases (ZFN) that target the BCL11A gene erythroid-specific enhancer (ESE) to increase endogenous HbF production in erythrocytes (RBC). It was formerly investigated for the treatment of beta-thalassemia, and it is currently being investigated for the treatment of sickle cell disease.
Indication
No indication information available.
Associated Conditions
No associated conditions information available.
Clinical Trials
FDA Approved Products
Singapore Approved Products
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