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Also known as: AAV vector containing porphobilinogen deaminase gene

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Company uniQure N.V.
DescriptionRecombinant adeno-associated viral (AAV) vector containing the porphobilinogen deaminase gene
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase I/II
Standard IndicationPorphyria
Indication DetailsTreat acute intermittent porphyria
Regulatory Designation

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