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Alnylam stock rises on early success with drug for rare genetic disorder

RNA interference (RNAi) technology is used to turn off disease-causing genes.
RNA interference (RNAi) technology is used to turn off disease-causing genes.Globe File photo/2013

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Alnylam Pharmaceuticals Inc. stock climbed after the Cambridge company reported positive early trial results for an experimental RNA interference drug, ALN-TTRsc. It’s designed to treat transthyretin familial amyloidosis, a rare genetic condition in which a starchy protein builds up in organs and tissues. ALN-TTRsc was evaluated for a form of the disease typically treated with a liver transplant. Alnylam will start a late stage-trial of the drug in 2014. It’s developing the drug in partnership with Genzyme.

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