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DTX401, DTX401 AAV G6Pase

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Company Dimension Therapeutics Inc.
DescriptionAdeno-associated viral (AAV) vector delivering glucose-6-phosphatase catalytic subunit-related protein (G6PC; G6Pase)
Molecular Target Glucose-6-phosphatase catalytic subunit-related protein (G6PC) (G6Pase)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationGlycosphingolipid storage disorders
Indication DetailsTreat glycogen storage disease type Ia (GSDIa)
Regulatory Designation U.S. - Orphan Drug (Treat glycogen storage disease type Ia (GSDIa));
EU - Orphan Drug (Treat glycogen storage disease type Ia (GSDIa))
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