Alnylam scientists and collaborators presented new pre-clinical data from our ALN-TTR program at the XII International Symposium on Amyloidosis in Rome on April 18 – 21, 2010. These data demonstrated, for the first time, that treatment with an RNAi therapeutic can result in regression of pre-existing pathogenic TTR deposits in peripheral tissues.
The new pre-clinical studies, performed in collaboration with Dr. Maria Saraiva at the Institute for Molecular and Cellular Biology in Portugal, used a transgenic mouse model where the mutant human V30M TTR gene is over-expressed. Specifically, the new data demonstrated that administration of ALN-TTR01 in mature transgenic mice resulted in the regression of existing pathogenic mutant human V30M TTR deposits in tissues, including: dorsal root ganglia, sciatic nerve, stomach, and intestines. These effects in diseased tissues were mediated through silencing of the mutant human TTR expressed in the liver, resulting in regression of established peripheral TTR deposits. ALN-TTR01 administration resulted in a greater than 90% regression of measurable TTR deposits as compared with control-treated animals.