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PRX-ASS1

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Company PhaseRx Inc.
DescriptionIntracellular enzyme replacement therapy
Molecular Target
Mechanism of ActionEnzyme replacement therapy
Therapeutic ModalityBiologic: Enzyme
Latest Stage of DevelopmentPreclinical
Standard IndicationUrea cycle disorder (UCD)
Indication DetailsTreat argininosuccinate synthase 1 deficiency
Regulatory Designation
Partner

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