The FDA granted orphan-drug status to Alnylam Pharmaceuticals' ALN-AT3 as a treatment for hemophilia A, just a week after receiving the same designation for hemophilia B. ALN-AT3, an injectable RNAi therapeutic designed to treat several rare bleeding diseases, is expected to enter Phase I human studies next year.
Alnylam's RNAi candidate wins orphan designation for hemophilia A
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