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AT001

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Collapse Summary General Information
Company Audentes Therapeutics Inc.
DescriptionAdeno-associated viral serotype8 (AAV8) vector encoding the myotubularin 1 (MTM1) gene
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard Indication Musculoskeletal (unspecified)
Indication DetailsTreat X-linked myotubular myopathy
Regulatory Designation

Partner

Genethon


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