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DTX301, DTX301 AAV OTC

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Company Dimension Therapeutics Inc.
Description 
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationUrea cycle disorder (UCD)
Indication DetailsTreat ornithine transcarbamylase deficiency (OTCD)
Regulatory Designation U.S. - Orphan Drug (Treat ornithine transcarbamylase deficiency (OTCD))
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