Alnylam Pharmaceuticals Phase I Clinical Study of ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR)

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). The study is aimed at evaluating the safety and tolerability of ALN-TTR02 in healthy volunteers. In addition, the study will evaluate the clinical activity of ALN-TTR02 based on measurements of serum levels of TTR, the disease-causing protein in patients with ATTR. ALN-TTR02 utilizes the company’s proprietary second-generation lipid nanoparticle (LNP) technology using the “MC3” lipid. Alnylam expects to present data from this study in the third quarter of 2012.

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