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rAAV1-CB-hAAT (AGTC-0106)

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Company Applied Genetic Technologies Corp.
DescriptionGene therapy using adeno-associated virus (AAV) to deliver alpha 1-antitrypsin (AAT) gene
Molecular Target Not applicable
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationAlpha-antitrypsin (AAT) deficiency
Indication DetailsTreat alpha-1 antitrypsin (AAT) deficiency
Regulatory Designation

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