Beyond knockdown

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...