Alnylam Forms Collaboration with Max Planck Institute to Investigate Biological Mechanisms for Cellular Uptake of RNAi Therapeutics

Company to Work with Leading Laboratory in Cell Trafficking Field as Part of Continued Commitment to Advance Research on Delivery of RNAi Therapeutics

CAMBRIDGE, MA, USA | August 11, 2008 | Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced it has formed an exclusive research agreement with the Max Planck Institute of Molecular Cell Biology and Genetics in Dresden, Germany, to investigate and characterize the molecular mechanisms underlying intracellular transport of small interfering RNA, (siRNA), the molecules that mediate RNAi.

Alnylam scientists will work closely with the laboratory of Marino Zerial, Ph.D., whose team is internationally recognized as being at the forefront of the intracellular trafficking field and have the needed expertise and capabilities to investigate cellular uptake pathways in a robust and high-throughput manner. Alnylam will have an option on any intellectual property that results from this agreement.

"Alnylam has a long-standing commitment to scientific excellence in the field of RNAi therapeutics and our new collaboration with Professor Zerial and his team is representative of the collaborations we have and will continue to form as we seek to increase our understanding of the underlying mechanism of cellular delivery of RNAi therapeutics," said Victor Kotelianski, M.D., Ph.D., Vice President for Research at Alnylam. "This agreement complements our existing delivery collaborations and will provide deep insights into the cellular uptake of siRNAs and the pathways by which they function. We believe the findings from this effort will allow us to further optimize and expand our broad range of delivery approaches for RNAi therapeutics."

"I am very excited to be working with Alnylam, a leader in the field of RNAi therapeutics, to advance our understanding of how siRNAs are taken up into the cell cytoplasm, where they act to silence disease-causing genes," said Marino Zerial, Ph.D., research group leader and director at the Max Planck Institute of Molecular Cell Biology and Genetics. "Through this collaboration, we will increase our understanding of how different siRNA formulations are internalized within cells and then use this knowledge to further optimize approaches for delivery of RNAi therapeutics."

About RNA Interference (RNAi)

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today. The Nobel Prize for Physiology or Medicine was awarded in 2006 to researchers in this field. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. RNAi therapeutics target the cause of diseases by potently silencing specific messenger RNAs (mRNAs), thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in fundamentally new ways.

About Alnylam Pharmaceuticals

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is applying its therapeutic expertise in RNAi to address significant medical needs, many of which cannot effectively be addressed with small molecules or antibodies, the current major classes of drugs. Alnylam is leading the translation of RNAi as a new class of innovative medicines with peer-reviewed research efforts published in the world's top scientific journals including Nature, Nature Medicine, and Cell. The company is leveraging these capabilities to build a broad pipeline of RNAi therapeutics; its most advanced program is in Phase II human clinical trials for the treatment of respiratory syncytial virus (RSV) infection. In addition, the company is developing RNAi therapeutics for the treatment of a wide range of disease areas, including hypercholesterolemia, liver cancers, and Huntington's disease. The company's leadership position in fundamental patents, technology, and know-how relating to RNAi has enabled it to form major alliances with leading companies including Medtronic, Novartis, Biogen Idec, Roche, Takeda, and Kyowa Hakko Kogyo. To reflect its outlook for key scientific, clinical, and business initiatives, Alnylam has established "RNAi 2010" which includes the company's plan to significantly expand the scope of delivery solutions for RNAi therapeutics, have four or more programs in clinical development, and to form four or more new major business collaborations, all by the end of 2010. Alnylam is a joint owner of Regulus Therapeutics LLC, a joint venture focused on the discovery, development, and commercialization of microRNA therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, visit

SOURCE: Alnylam Pharmaceuticals

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