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ALN-AAT

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Company Alnylam Pharmaceuticals Inc.
DescriptionShort interfering RNA (siRNA) targeting alpha 1-antitrypsin (AAT; A1AT; SERPINA1) conjugated to GalNAc ligand
Molecular Target Alpha 1-antitrypsin (AAT) (A1AT) (SERPINA1)
Mechanism of ActionGene silencing
Therapeutic ModalityNucleic acid: Linear RNA: siRNA
Latest Stage of DevelopmentPreclinical
Standard IndicationLiver disease
Indication DetailsTreat liver disease caused by alpha 1-antitrypsin (AAT; A1AT; SERPINA1) deficiency
Regulatory Designation

Partner


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