Alnylam and GlaxoSmithKline Form Collaboration on RNAi Technology for Vaccine Production

Alnylam and GlaxoSmithKline Form Collaboration on RNAi Technology for Vaccine Production

- Alnylam VaxiRNATM Platform Applies RNAi to Enhance Vaccine Production for Influenza and Other Pathogens -

CAMBRIDGE, Mass., Nov 01, 2011 (BUSINESS WIRE) --

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today it has formed a collaboration with GlaxoSmithKline (GSK) focused on Alnylam's VaxiRNATM technology for certain GSK vaccine products, including influenza. VaxiRNA is a new RNAi technology for the enhanced production of viruses used in the manufacture of vaccine products. GSK, a leading vaccine manufacturer, is the first company to form a collaboration accessing this new platform.

"At Alnylam, we continue to pioneer innovative opportunities for our RNAi platform that could have transformative impact in medicine and the biopharmaceutical industry. This is exemplified with VaxiRNA, where siRNA technology is being used to increase virus titers in the production of vaccine products. Since production of vaccines can be limiting for the scale and speed of global immunization against many pathogens, including influenza, we believe that VaxiRNA can become an enabling technology for the broader vaccine industry," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "As we advance our VaxiRNA effort, we are excited to form a new collaboration with GSK, a global leader and innovator in vaccine development and manufacturing. With GSK, we aim to apply VaxiRNA technology to enhance influenza vaccine production in cell culture, where Alnylam will receive research funding, in addition to potential milestones and payments on unit sales of commercialized vaccine product."

Alnylam's VaxiRNA platform applies siRNAs for the silencing of specific genes that limit or prevent efficient growth of viruses in vaccine manufacturing systems, including those in cell culture and also in eggs. The VaxiRNA platform stems from work Alnylam has performed as part of its Alnylam Biotherapeutics efforts, where RNAi technology has been applied to improve the manufacture of biotherapeutic products, such as recombinant proteins and monoclonal antibodies. This new platform addresses the significant unmet commercial need for innovative technologies that can improve the manufacture of vaccine products, especially where vaccine production is a limiting factor for the scale and speed of global immunization needs.

The GSK collaboration is focused initially on influenza vaccine production in cell culture systems. Under the terms of the agreement, GSK will provide funding and certain milestone payments to Alnylam. If successfully applied in the manufacture of commercial product, Alnylam will receive payments on unit product sales. In addition, GSK has obtained an option for VaxiRNA applications toward two additional vaccine products.

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 VaxiRNA(TM)

Alnylam is applying RNAi technology to improve the manufacturing processes for vaccines in an effort called VaxiRNA. With VaxiRNA, Alnylam is using siRNAs that silence specific genes in vaccine production systems, such as cells or eggs, which limit or prevent the efficient growth of viruses used in the manufacture of vaccine products. New innovations in vaccine manufacturing are needed to enable the scale and speed of global immunization for a number of pathogens. In 2011, Alnylam formed a VaxiRNA partnership with GlaxoSmithKline for influenza vaccine production.

About Alnylam Pharmaceuticals

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, and ALN-APC for the treatment of hemophilia. As part of its "Alnylam 5x15TM" strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development 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 application in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam's VaxiRNA(TM) 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

Alnylam Forward-Looking Statements

Various statements in this release concerning Alnylam's future expectations, plans and prospects, including without limitation, Alnylam's views with respect to the potential for VaxiRNA to apply RNAi technology to improve the manufacturing process for vaccines, its expectations regarding the receipt of research funding, milestone payments and payments on unit product sales under its collaboration with GlaxoSmithKline, as well as 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 successfully apply RNAi technology to improve the manufacturing process for vaccines, including in connection with its GSK collaboration, demonstrate the efficacy and safety of its drug candidates in pre-clinical and human clinical trials, and establish and maintain strategic business alliances and new business initiatives, 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.

SOURCE: Alnylam Pharmaceuticals, Inc.

Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Senior Director, Investor Relations and
Corporate Communications
Amanda Sellers (Media), 202-955-6222 x2597