The FDA granted an orphan-drug designation to Alnylam Pharmaceuticals' ALN-AT3 as a possible treatment for hemophilia B. The drug, based on the firm's RNAi gene-silencing technology, is also under development for hemophilia A and other bleeding disorders. Alnylam plans to submit an investigational new drug application for ALN-AT3 this year and begin an early-stage trial next year.

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