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Patisiran, an RNA interference drug being developed by Alnylam and Sanofi, met its primary goals in a Phase III study as a treatment for hereditary ATTR amyloidosis, a rare genetic disease. Alnylam plans to file a New Drug Application later this year. “We are proud to report the first-ever positive Phase III results for an RNAi therapeutic,” says Alnylam CEO John Maraganore. Alnylam’s stock price soared more than 20% on the news. The firm earlier experienced setbacks for two other RNAi drugs, revusiran and fitusiran.
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