Alnylam Pharmaceuticals, Inc.
Call it The Silence of the Genes. Alnylam Pharmaceuticals runs interference with RNA to prevent disease. RNA interference (RNAi) technology developed by the biotech firm can selectively shut off harmful genes. The company is developing a pipeline of candidates both individually and through collaborations with other drugmakers. Its disease targets include rare genetic disorder ATTR (transthyretin-mediated amyloidosis) and RSV (respiratory syncytial virus), a common childhood viral infection. Additional R&D programs target neurological disorders, cardiovascular conditions, and cancers. Alnylam has R&D alliances and licensing agreements with companies including Roche, Biogen Idec, and Takeda Pharmaceuticals.