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AT001

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Collapse Summary General Information
Company Audentes Therapeutics Inc.
DescriptionAn adeno-associated virus (AAV) 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


 Product Details
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