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AADC gene therapy, AGIL-AADC (formerly AADC)

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Company National Taiwan University Hospital
DescriptionAdeno-associated viral (AAV) vector encoding the dopa decarboxylase (DDC; AADC) gene
Molecular Target Dopa decarboxylase (DDC) (AADC)
Mechanism of Action 
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase II
Standard IndicationCentral nervous system (CNS) disorders
Indication DetailsTreat AADC deficiency
Regulatory Designation U.S. - Rare Pediatric Disease (Treat AADC deficiency)
PartnerAgilis Biotherapeutics LLC

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