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Genzyme will pay Alnylam Pharmaceuticals $22.5 million for the right to develop RNAi-based treatments for transthyretin-mediated amyloidosis (ATTR) in Japan and other Asian countries. ATTR is an inherited disease of the nervous system and heart caused by mutations in the transthyretin protein; a significant number of people in Japan are susceptible to it. Alnylam has two therapies that inhibit the protein: ALN-TTR02, in Phase II studies, and ALN-TTRsc, in preclinical development.
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