Name: Alnylam U.S., Inc.
Address: 300 3rd St Ste 3, Cambridge, MA, 02142-1106, USA
Telephone: (617) 551-8200

Alnylam is developing an entirely new class of innovative medicines based on a breakthrough discovery in biology known as RNA interference, or RNAi. With RNAi technology, we have the opportunity to treat disease and impact the lives of patients in a fundamentally new way by silencing disease-causing genes upstream of today's medicines.

(al-NIGH-lam)
Located in the constellation Orion, Alnylam is the name of the center star of Orion's belt. The star has a luminosity that is 250,000 times greater than the sun, representative of the potential strength that RNAi therapeutics could bring to bear for human health.

As scientists continue to investigate the human genome, where we now have the complete genetic sequence, there have been remarkable developments in the understanding of disease and consequently a large increase in the number of molecular disease targets to impact a broad range of human disease. Paradoxically, with all these new opportunities to significantly impact disease, the challenge for the industry has been that today's therapeutic modalities, such as small molecules and monoclonal antibodies cannot access most (~70%-80%) of these new disease targets. With RNAi, we can target virtually any gene in the genome involved in the causal pathway of disease. The possibility of targeting these previously "undruggable" targets with RNAi is transformative for new drug discovery.

We have made very significant strides in the development and advancement of RNAi therapeutics. Recent months represent a remarkable period of clinical accomplishments for Alnylam's pipeline efforts where we demonstrated RNAi proof of mechanism in biopsy samples from our ALN-VSP liver cancer program, showed clear human proof of concept in patients with robust silencing of a disease gene in our ALN-TTR program, and documented first-ever clinical efficacy for RNAi therapeutics in our ALN-PCS program. Indeed, we believe these achievements greatly strengthen our "Alnylam 5x15" product strategy, and we are more confident than ever in our ability to harness the RNAi pathway for the development of high impact, innovative medicines.

As we effect our transformation from a platform company to a product company, our near-term efforts will focus on what we believe to be our highest value opportunities with accelerated clinical development plans. Specifically, we aim to maximally apply Alnylam resources to our programs in transthyretin-mediated amyloidosis (link to TTR page) and hemophilia (link to APC page), while advancing our other multiple pipeline programs through existing alliances and new partnerships.

Alnylam U.S., Inc.

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