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Feature Story

Beyond knockdown

Alnylam's patisiran data in TTR-mediated amyloidosis validate siRNA platform

By Erin McCallister, Senior Editor

New Phase II data from Alnylam Pharmaceuticals Inc. are the first to validate the hypothesis on which the company was founded, by showing a clinical benefit in patients with a rare nerve disorder who were treated with the company's lead siRNA candidate, patisiran. Proof-of-concept data for another program could come before year end.

Last week, Alnylam announced preliminary data from an ongoing open-label Phase II extension study of patisiran in a form of transthyretin (TTR)-mediated amyloidosis called familial polyneuropathy (FAP). Patisiran is an RNAi therapeutic targeting the TTR gene.

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