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Lenti-D gene therapy

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Company bluebird bio Inc.
DescriptionHematopoietic stem cell therapy with a lentiviral vector encoding the wild-type gene that encodes the adrenoleukodystrophy (ALD) protein
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Lentivirus
Latest Stage of DevelopmentPhase II/III
Standard IndicationLeukodystrophy
Indication DetailsTreat childhood cerebral adrenoleukodystrophy; Treat X-linked adrenoleukodystrophy (ALD)
Regulatory Designation


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