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Autologous CD34+ cells

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Company Fondazione Telethon
DescriptionAutologous CD34+ cells transfected with a lentiviral vector encoding functional Wiskott-Aldrich syndrome protein
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Lentivirus
Latest Stage of DevelopmentPhase I/II
Standard Indication Hematology (unspecified)
Indication DetailsTreat Wiskott-Aldrich syndrome
Regulatory Designation

EU - Orphan Drug (Treat Wiskott-Aldrich syndrome)

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