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ISIS-TTRRx

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Company Isis Pharmaceuticals Inc.
DescriptionTTR antisense inhibitor
Molecular Target Transthyretin (TTR)
Mechanism of ActionAntisense therapy
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentPhase III
Standard IndicationAmyloidosis
Indication DetailsTreat transthyretin (TTR) amyloidosis; Treat transthyretin (TTR) familial amyloid polyneuropathy (FAP)
Regulatory Designation

U.S. - Fast Track (Treat transthyretin (TTR) familial amyloid polyneuropathy (FAP));
U.S. - Fast Track (Treat transthyretin (TTR) amyloidosis);
U.S. - Orphan Drug (Treat transthyretin (TTR) amyloidosis);
EU - Orphan Drug (Treat transthyretin (TTR) amyloidosis)

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