Alnylam and UMass Medical School announce Tuschl I patent upheld in European opposition proceedings

Cambridge, Mass., and Worcester, Mass., March 1, 2012 – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and the University of Massachusetts Medical School (UMMS) reported today that the European Patent Office (EPO) has upheld the Tuschl I '726 patent (EP 1309726) in oral opposition proceedings held in Munich, Germany. The requested claims of the '726 patent were upheld without any modification. Opponents included Sanofi-Aventis Deutschland GmbH, Silence Therapeutics AG, and BASF SE.

Inventors on the patent are David P. Bartel, PhD, a Howard Hughes Medical Institute Investigator and professor of biology at the Massachusetts Institute of Technology; Phillip A. Sharp, PhD, Institute Professor at the Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology and a 1993 Nobel Laureate; Thomas Tuschl, PhD, a Howard Hughes Medical Institute Investigator and professor at Rockefeller University; and Phillip D. Zamore, PhD, a Howard Hughes Medical Institute Investigator and the Gretchen Stone Cook Professor of Biomedical Sciences at the University of Massachusetts Medical School, where he co-directs the RNA Therapeutics Institute.

"The Tuschl family of patents defines key discoveries central to the advancement of RNAi therapeutics to patients," said James P. McNamara, PhD, executive director, Office of Technology Management, University of Massachusetts Medical School. "The Tuschl I patent is a critical invention by Professors Tuschl, Zamore, Bartel, and Sharp regarding the RNAi mechanism. We are pleased to see this patent fully upheld in Europe in these opposition proceedings."

"We are very pleased with the outcome of these opposition proceedings which resulted in the claims from the Tuschl I '726 patent being fully upheld. This decision by the EPO affirms our belief in the validity of these claims, and the novelty of the Tuschl I invention, and supports the relevance of Tuschl I for the development and commercialization of RNAi therapeutics," said Laurence Reid, PhD, senior vice president and chief business officer of Alnylam. "Alnylam continues to leverage its leading patent estate for the advancement of innovative medicines to patients and also continues to enable the entire RNAi therapeutics field with over 30 license agreements formed to date."

"The research discoveries that form the basis for this patent were a significant milestone in the explication of the basic science of RNAi and the path to the world of RNA therapeutics," said Zamore. "The affirmation of the intellectual basis of these discoveries by the EPO will accelerate the opportunities for laboratories and research organizations around the world to use this science to work toward therapeutics to treat human disease."

Alnylam is the co-exclusive licensee of the Tuschl I '726 patent which is owned by the Max Planck Society, the Massachusetts Institute of Technology, the Whitehead Institute for Biomedical Research, and the University of Massachusetts Medical School. This patent is a key component to Alnylam's fundamental intellectual property estate that comprises numerous issued or granted patents and a large number of pending patent applications that together broadly cover RNAi therapeutics, including small interfering RNAs, or siRNAs, the molecules that mediate RNAi. The Tuschl I '726 patent consists of 14 claims broadly covering RNAi methods, including methods of reducing the expression of a gene, with double stranded RNAs between 21 and 23 nucleotides in length of mammalian or viral origin. The patent also includes claims covering methods of examining the function of a gene, as well as the use of both unmodified and chemically modified double stranded RNAs.

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.

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, ALN-APC for the treatment of hemophilia, and ALN-TMP for the treatment of hemoglobinopathies. As part of its "Alnylam 5x15TM" strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in clinical development, including programs in advanced stages, on its own or with a partner by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington's disease. The company's leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for applications in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam's VaxiRNA™ platform applies RNAi technology to improve the manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world's top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, statements regarding Alnylam's expectations with respect to its "Alnylam 5x15" product strategy, 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, without limitation, Alnylam's ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, as well as those risks more fully discussed in the "Risk Factors" section of its most recent annual report on Form 10-K 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.

The University of Massachusetts Medical School, one of the fastest growing academic health centers in the country, has built a reputation as a world-class research institution, consistently producing noteworthy advances in clinical and basic research. UMMS attracted more than $277 million in research funding in 2011, 80 percent of which comes from federal funding sources. The mission of UMass Medical School is to advance the health and well-being of the people of the commonwealth and the world through pioneering education, research, public service and health care delivery with its clinical partner, UMass Memorial Health Care. For more information, visit www.umassmed.edu.