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ALN-AS1

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Company Alnylam Pharmaceuticals Inc.
DescriptionSubcutaneous RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1)
Molecular Target Aminolevulinate synthase-1 (ALAS-1)
Mechanism of ActionGene silencing
Therapeutic ModalityNucleic acid: Linear RNA
Latest Stage of DevelopmentPreclinical
Standard IndicationPorphyria
Indication DetailsTreat acute intermittent porphyria; Treat hepatic porphyrias
Regulatory Designation

Partner


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