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patisiran (ALN-TTR02, GENZ438027, GZ438027)

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Company Alnylam Pharmaceuticals Inc.
DescriptionRNAi therapeutic targeting the transthyretin (TTR) gene using second-generation lipid nanoparticle technology
Molecular Target Transthyretin (TTR)
Mechanism of ActionGene silencing
Therapeutic ModalityNucleic acid: Linear RNA: microRNA
Latest Stage of DevelopmentPhase III
Standard IndicationAmyloidosis
Indication DetailsTreat familial amyloidotic polyneuropathy (FAP); Treat transthyretin (TTR)-mediated amyloidosis
Regulatory Designation U.S. - Fast Track (Treat familial amyloidotic polyneuropathy (FAP));
U.S. - Orphan Drug (Treat familial amyloidotic polyneuropathy (FAP))

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