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.