Alnylam, Collaborators Publish Preclinical Data on RNAi for Porphyria | GenomeWeb

NEW YORK (GenomeWeb) – Researchers from Mount Sinai School of Medicine, along with collaborators from Alnylam Pharmaceuticals, reported this week new animal data showing that RNAi can be used to prevent and treat the neurovisceral attacks that characterize acute intermittent porphyria (AIP).

The findings, which provide additional proof of concept for Alnylam's preclinical AIP treatment ALN-AS1, are based on experiments using siRNAs formulated in lipid nanoparticles and show that such agents are effective at doses as low as 1 mg/kg.

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