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Alnylam Releases Preclinical Data Showing Efficacy of TTR Amyloidosis Rx Candidate


Alnylam Pharmaceuticals this week released new preclinical data showing that its TTR amyloidosis drug candidate could trigger dose-dependent reductions of liver TTR messenger RNA and serum TTR protein levels by more than 80 percent in transgenic mice and non-human primates.

The data also demonstrated that the silencing effects extended more than three weeks after a single dose administration, according to the company.

TTR amyloidosis is a hereditary, systemic disease caused by a mutation in the transthyretin gene. This summer, Alnylam announced that it had moved its investigational TTR amyloidosis therapeutic, ALN-TTR, into its formal drug-development pipeline (see RNAi News, 8/13/2009).

Alnylam said that it expects to begin clinical testing of the drug in early 2010.