BIO Ventures for Global Health Chosen as Administrator of Intellectual Property Pool Involving GSK and Alnylam as Participants

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and GlaxoSmithKline (GSK), announced today that they have chosen BIO Ventures for Global Health (BVGH) to administer an Intellectual Property (IP) Pool that was established in February 2009. The IP Pool was formed to aid in the discovery and development of new medicines for the treatment of certain neglected tropical diseases, defined by the U.S. Food and Drug Administration (FDA), in the world’s least developed countries. By adopting a more accessible approach to IP, the pool facilitates access to compounds and technologies and, most importantly, industrial know-how for organizations that want to conduct research on treatments for these neglected diseases.

"We are delighted that BVGH has agreed to serve as administrator of this critical initiative. The more companies, academic institutions, and foundations that join the pool, the more effective it will be, and we are confident that BVGH’s role will help grow participation,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We at Alnylam share GSK’s and BVGH’s commitment to the advancement of innovative medicines to patients, so we cannot ignore the potential of our RNAi technology to make a difference in the discovery of important new medicines for neglected diseases that afflict millions of people each year.”

"New medicines for diseases that primarily affect the poor in developing countries have been slow in coming because although the market is large in numbers, most of the people affected and their governments cannot afford to pay even moderate prices. This means that much of the development of new medicines is taking place in innovative non-profits and academic research centers,” says Melinda Moree, Chief Executive Officer of BIO Ventures for Global Health, an independent, non-profit organization. "For many of these academic and global health non-profit groups, however, intellectual property can be perceived as preventing rather than enabling their work. I am excited to use this new role to help those working on developing products for neglected diseases speed up their efforts by accessing the patents, technologies, and product development expertise available in the IP Pool.”

As the pool’s administrator, BVGH will organize disease-specific meetings that identify the gaps in expertise and intellectual property that currently exist in product development for neglected diseases. BVGH will then help global health researchers work with industry to fill these gaps so that the resources generously made available by companies will be used to create medicines for neglected diseases faster and more efficiently.

Through the patent pool, Alnylam has contributed its RNAi intellectual property, technology and know-how on a royalty-free, non-profit basis in the least developed countries via licensing agreements with qualified third parties. Such organizations will be engaged in research efforts focused on discovery of new medicines for neglected tropical diseases and their distribution to least developed countries. In the near term, Alnylam RNAi technology is expected to help validate novel drug targets for the discovery and development of treatments for the targeted diseases in least developed countries. For example, the technology has already helped to identify new targets for malaria treatments.^1,2,3 In the future, RNAi therapeutics may themselves be developed and used directly in the treatment of more neglected tropical diseases.

About the Intellectual Property (IP) Pool

The diseases targeted by the pool are the 16 diseases identified by the FDA for its own Neglected Tropical Diseases initiative. These are tuberculosis, malaria, blinding trachoma, buruli ulcer, cholera, dengue/dengue haemorrhagic fever, racunculiasis, fascioliasis, human African trypanosomiasis, leishmaniasis, leprosy, lymphatic filariasis, onchocerciasis, schistosomiasis, soil transmitted helminthiasis and yaws. The geographic focus of the pool will be the world’s Least Developed Countries as identified by the United Nations and includes much of western and central Africa as well as several countries in Southeast Asia.

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 which was awarded the 2006 Nobel Prize for Physiology or Medicine. 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. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.

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 and is partnered with Cubist and Kyowa Hakko Kirin. In addition, the company is developing RNAi therapeutics for the treatment of a wide range of disease areas, including liver cancers, TTR-mediated amyloidosis, hypercholesterolemia, 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, Kyowa Hakko Kirin, and Cubist. Alnylam and Isis are joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development, and commercialization of microRNA-based therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

About BIO Ventures for Global Health

BIO Ventures for Global Health is a non-profit organization whose mission is to save lives by accelerating the development of novel biotechnology-based drugs, vaccines, and diagnostics to address the unmet medical needs of the developing world. The organization spurs biotech industry involvement in global health product development by increasing biotech and global health partnerships, designing and advocating for compelling market-based incentives, and synthesizing and disseminating critical information and quantitative analysis. For more information, please visit www.bvgh.org.

About GlaxoSmithKline – one of the world’s leading research-based pharmaceutical and healthcare companies – is committed to improving the quality of human life by enabling people to do more, feel better and live longer. For further information, please visit www.gsk.com.

Alnylam Forward-Looking Statement

Various statements in this release concerning Alnylam’s future expectations, plans and prospects, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including the company’s ability to successfully prosecute and enforce its patents around the world, as well as those risks more fully discussed in the "Risk Factors” section of its most recent quarterly report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.

References

¹ Prudêncio et al. (2008) Kinome-Wide RNAi Screen Implicates at Least 5 Host Hepatocyte Kinases in Plasmodium Sporozoite Infection, PLoS Pathog 4(11): e1000201. doi:10.1371/ journal. ppat.1000201

² Rodrigues et al. (2008) Host Scavenger Receptor SR-BI Plays a Dual Role in the Establishment of Malaria Parasite Liver Infection, Cell Host & Microbe 4, 271-282

³ Epiphanio et al. (2008) Heme Oxygenase-1 Is an Anti-Inflammatory Host Factor that Promotes Murine Plasmodium Liver Infection, Cell Host & Microbe, 3: 331-338