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Company Alnylam Pharmaceuticals Inc.
DescriptionSubcutaneous RNAi therapeutic targeting glycolate oxidase using the GalNAc-conjugate delivery platform
Molecular Target Glycolate oxidase
Mechanism of ActionGene silencing
Therapeutic ModalityNucleic acid: Linear RNA: siRNA
Latest Stage of DevelopmentPhase I/II
Standard IndicationHyperoxaluria
Indication DetailsTreat primary hyperoxaluria type 1 (PH1)
Regulatory Designation U.S. - Orphan Drug (Treat primary hyperoxaluria type 1 (PH1));
EU - Orphan Drug (Treat primary hyperoxaluria type 1 (PH1))

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