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Alnylam Pharmaceuticals
RNAi technology
Alnylam is founded by a group of academic bioscientists: Phillip Sharp, Paul Schimmel, and Dave Bartel of the Massachusetts Insititute of Technology, Tom Tuschl of the Max Planck Institute for Biophysical Chemistry in Göttingen, Germany, and Phil Zamore of the University of Massachusetts Medical School, along with entrepreneur Cristoph Westphal. This stellar group secures funding from an investment syndicate composed of leading venture capital providers including Polaris Ventures, Atlas Venture, ARCH Capital Partners, and Cardinal Partners. The company is set up in Cambridge, Massachusetts to develop RNA interference (RNAi) therapeutics.
RNA interference refers to a naturally-occurring intracellular process in which small 'interfering' RNA molecules (siRNA) binds and cleaves messenger RNA molecules, preventing them from delivering their messages to ribosomes, the cellular factories in which proteins are assembled according to the transcribed and translated genetic code. The actions of siRNAs disrupt protein synthesis. Alnylam's long-term goal is to harness this natural cellular technology for the production of “important nucleic acid-based drugs that...inhibit the production of pathological proteins that ultimately result in cancer, infectious diseases, inflammation, and other disorders.”
The company has a broad pipeline, and has demonstrated the possibility of RNAi interventions in human clinical trials. Alnylam is currently developing or testing systems that deliver molecular therapies for amyloidosis, hypercholesterolemia, anemia, hemophilia, and Huntington's Disease. The firm's most advanced project, for respiratory syncytial virus (RSV) infection, has entered phase II clinical trials. A liver cancer drug is also perched on the threshold of phase II testing.
Alnylam headquarters, Cambridge, MA
