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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 ornithine transcarbamylase deficiency (OTCD)
Regulatory Designation U.S. - Orphan Drug (Treat ornithine transcarbamylase deficiency (OTCD))

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