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AT982 (formerly AT002)

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Company Audentes Therapeutics Inc.
DescriptionAdeno-associated viral serotype 9 (AAV9) vector carrying the acid alpha glucosidase (GAA) gene
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationPompe's disease
Indication DetailsTreat Pompe's disease
Regulatory Designation
Partner

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