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ALN-TTRsc

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Company Alnylam Pharmaceuticals Inc.
DescriptionSubcutaneous formulation of an RNAi therapeutic targeting the transthyretin (TTR) gene using the GalNAc-conjugate delivery platform
Molecular Target Transthyretin (TTR)
Mechanism of ActionGene silencing
Therapeutic ModalityNucleic acid: Linear RNA
Latest Stage of DevelopmentPhase II
Standard IndicationAmyloidosis
Indication DetailsTreat familial amyloidotic cardiomyopathy (FAC); Treat transthyretin (TTR)-mediated amyloidosis; Treat TTR cardiac amyloidosis
Regulatory Designation

Partner

Sanofi


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