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LentiGlobin HPV569 gene therapy

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Company bluebird bio Inc.
DescriptionAutologous CD34+ stem cells transduced ex vivo with a lentiviral vector delivering the human beta globin gene
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Lentivirus
Latest Stage of DevelopmentPhase I/II
Standard IndicationThalassemia
Indication DetailsTreat sickle cell anemia and beta-thalassemia
Regulatory Designation

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