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Patisiran, a second-generation siRNA therapy that targets the transthyretin (TTR) gene, for the treatment of TTR-mediated amyloidosis (ATTR). Patisiran comprises siRNA molecules encapsulated in lipid nanoparticles, using Tekmira Pharmaceuticals’ lipid nanoparticle technology, to enable delivery into the cell.
TTR is a soluble transport protein. Mutations in the TTR gene, which is predominantly expressed in the liver, cause TTR to form abnormal insoluble amyloid proteins which accumulate and damage organs and tissue, mainly the heart or nerves. The condition is referred to as familial amyloid polyneuropathy (FAP) when peripheral nerves are primarily affected, and familial amyloid cardiomyopathy (FAC) in the case of cardiac presentations.
For further details - http://investors.alnylam.com/releasedetail.cfm?ReleaseID=805999
Adapted from - Thomson Reuters Cortellis Competitive Intelligence