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Company Alnylam Pharmaceuticals Inc.
DescriptionOnce-quarterly subcutaneous RNAi therapeutic that targets transthyretin (TTR) mRNA and uses the Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA conjugate delivery platform
Molecular Target Transthyretin (TTR) mRNA
Mechanism of ActionGene silencing
Therapeutic ModalityNucleic acid: Linear RNA: siRNA
Latest Stage of DevelopmentPhase I
Standard IndicationAmyloidosis
Indication DetailsTreat TTR-mediated amyloidosis
Regulatory Designation

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