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VY-FXN01

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Collapse Summary General Information
Company Voyager Therapeutics Inc.
DescriptionAdeno-associated virus (AAV) -based gene therapy designed to deliver the frataxin (FXN; FRDA) gene
Molecular Target Frataxin (FXN) (FRDA)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationAtaxia
Indication DetailsTreat Friedreich's ataxia (FRDA)
Regulatory Designation

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