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Autologous CD34+ cells

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Company Fondazione Telethon
DescriptionAutologous CD34+ cells transfected with a lentiviral vector encoding functional arylsulfatase A (ARSA) protein
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Lentivirus
Latest Stage of DevelopmentPhase I/II
Standard IndicationLeukodystrophy
Indication DetailsTreat metachromatic leukodystrophy (MLD)
Regulatory Designation EU - Orphan Drug (Treat metachromatic leukodystrophy (MLD))

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