Alnylam Biotherapeutics Presents New Data on RNAi Applications in Biotherapeutics Manufacturing

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Biotherapeutics, a division of Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the presentation of new data at the IBC 6^th Annual Cell Line Development and Engineering conference held in San Francisco, California from June 21 – 24, 2010. Alnylam established Alnylam Biotherapeutics to focus on the application of RNAi technology in manufacturing processes for biotherapeutics. The new results describe the discovery of highly effective novel delivery lipids (NDLs) that efficiently deliver siRNAs to manufacturing cell lines with no measurable adverse effects on cell density or viability. Furthermore, the NDLs demonstrate durable target gene silencing following a single dose, improved protein quality, and scalability to at least 40 liters.

“Our efforts in Alnylam Biotherapeutics are continuing to advance at a very rapid pace, clearly demonstrating the potential of RNAi technology to broadly and significantly improve the quality, quantity, and attributes of biotherapeutics.”

“The new research presented describes the discovery of proprietary delivery lipids that are superior to commercially available lipids in their cell tolerability and efficacy. Importantly, we have now demonstrated the application of RNAi technology with CHO cells grown in 40 liter bioreactors, with effects comparable to what we observed in one and three liter cell cultures,” said Stuart Pollard, Ph.D., Vice President, Scientific and Business Strategy of Alnylam. “Our efforts in Alnylam Biotherapeutics are continuing to advance at a very rapid pace, clearly demonstrating the potential of RNAi technology to broadly and significantly improve the quality, quantity, and attributes of biotherapeutics.”

Studies were performed using Alnylam’s proprietary NDLs, compared with commercially available lipids, to deliver siRNAs into Chinese Hamster Ovary (CHO) cells. Results showed:

improved tolerability for NDLs compared with commercially available lipids, with no measurable adverse effects on CHO cell growth rates or cell viability;
efficacy of target gene silencing, specifically a greater than 80% reduction in CHO cell lactate dehydrogenase activity, following a single dose administration of an siRNA with effects lasting over one week;
scalability of RNAi technology with comparable target gene silencing activity in CHO cell cultures grown in one, three, and 40 liter bioreactors; and,
ability to alter attributes of a recombinant monoclonal antibody, specifically a greater than 85% reduction in fucosylation, by targeting CHO cell glycosylation pathways.

Alnylam Biotherapeutics is advancing RNAi technologies to improve the quantity and quality of biologics manufacturing processes in mammalian cell culture, such as CHO cells, with the aim of transforming the $100 billion biologics marketplace comprised of recombinant proteins and monoclonal antibodies. In this application, RNAi technology can be applied to the improvement of manufacturing processes for existing marketed drugs, new drugs in development, and for the emerging biosimilars market. Alnylam has developed proprietary delivery lipids that enable efficient delivery of siRNAs into CHO cells as well as other cell systems that are used for the manufacture of biologics.

As Alnylam Biotherapeutics advances the technology, it plans to form partnerships with established biologics manufacturers, selling licenses, products, and services.

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 for the treatment of a wide range of disease areas, including respiratory syncytial virus (RSV), liver cancers, TTR-mediated amyloidosis (ATTR), hypercholesterolemia, and Huntington’s disease. In addition, Alnylam formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in manufacturing processes for biotherapeutic products, including recombinant proteins and monoclonal antibodies. 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 therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

Alnylam Forward-Looking Statement

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 Alnylam Biotherapeutics to develop RNAi technologies for application in manufacturing processes for biotherapeutic products, including recombinant proteins and monoclonal antibodies, 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 the company’s ability to continue to develop and advance RNAi technologies for biologics manufacturing, and to form partnerships with established biologics manufacturers, selling licenses, products, and services, 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.