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Company Errant Gene Therapeutics LLC
DescriptionAutologous hematopoietic stem cells transduced with lentiviral vector carrying the human beta-globin gene
Molecular Target
Mechanism of Action 
Therapeutic ModalityCell therapy: Stem cell
Latest Stage of DevelopmentPreclinical
Standard IndicationThalassemia
Indication DetailsTreat thalassemia
Regulatory Designation

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