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fitusiran (ALN-AT3)

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Company Alnylam Pharmaceuticals Inc.
DescriptionRNAi therapeutic targeting anti-thrombin III (AT3; SERPINC1) mRNA
Molecular Target Anti-thrombin III (AT3) (SERPINC1)
Mechanism of ActionGene silencing
Therapeutic ModalityNucleic acid: Linear RNA
Latest Stage of DevelopmentPhase II
Standard IndicationHemophilia
Indication DetailsTreat hemophilia; Treat hemophilia A; Treat hemophilia B
Regulatory Designation U.S. - Orphan Drug (Treat hemophilia B);
U.S. - Orphan Drug (Treat hemophilia A);
EU - Orphan Drug (Treat hemophilia)

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