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2004 World Technology Awards Winners & Finalists

Alnylam Pharmaceuticals

Please describe the work that you are doing that you consider to be the most innovative and of the greatest likely long-term significance.

Alnylam Pharmaceuticals is the leading company in the emerging field of RNA interference (RNAi) which represents one of the most promising new frontiers in drug discovery. Recent breakthroughs in understanding RNA's extensive role in essential cellular behaviors have opened up the potential for a whole new class of drugs based on RNAi.

Growing from its foundation as the world's first company focused on RNAi-based therapeutics, Alnylam is pioneering the effort to develop and commercialize therapeutic products based on RNAi for a broad range of therapeutic indications. The company's leadership in the field of RNAi is supported by its preeminent founders and advisors and its strengths in the fundamental patents, technology, and know-how that underlie the commercialization of RNAi-based therapeutics.

Over the last several years, in work done by Alnylam founders and others, RNAi has been identified as a fundamental cellular mechanism for gene silencing. Very recently, leveraging a series of important scientific breakthroughs, the possibility of targeting RNA as a tool in drug development has emerged. This revolutionary approach offers incredibly broad promise, as drugs can potentially be targeted "upstream" of protein synthesis in a natural way that is available in all cells. The discovery of RNAi has been has been heralded by many as a major breakthrough, and the journal Science named RNAi the top scientific achievement of 2002 as well as one of the top ten scientific advances of 2003.

Alnylamís founding scientists have shown that tiny snippets of RNA with two strands instead of the usual one can be used to selectively silence specific genes. Because many diseases are caused by the inappropriate activity of specific genes, silencing genes selectively through RNAi could provide a means to treat a wide range of human diseases. The underlying biological mechanism of RNAi was recently discovered, in part, by the scientific founders of Alnylam, who showed that RNAi is mediated by a molecule known as a small interfering RNA, or siRNA, and that chemically-synthesized siRNAs made in the laboratory can be introduced into cells and silence the activity of specific genes. Alnylam is developing chemically-synthesized siRNAs as potential drugs for a variety of diseases.

Alnylam's focus is to discover, develop, and commercialize therapeutic products based on RNAi for a broad range of therapeutic indications including viral, oncologic, metabolic, CNS and autoimmune diseases. The company's mission is to lead research in the field of RNAi, to build the leading pipeline of RNAi-based therapeutics, and to emerge as business leaders. The companyís stock is listed on the NASDAQ under the symbol ALNY and its global headquarters are in Cambridge, Massachusetts.