Health & Fitness
Health and Fitness
Health and Fitness
Fri, November 19, 2010
Thu, November 18, 2010
Alnylam Biotherapeutics Presents Data on New Applications of RNAi Technology for Biologics Manufacturing
CAMBRIDGE, Mass.--([ BUSINESS WIRE ])--Alnylam Biotherapeutics, a division of Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, presented new data at the MIT Center for Biomedical Innovationa™s Biomanufacturing Summit from its platform efforts aimed at developing RNAi technologies for applications in biologics manufacturing. The new studies show that siRNAs designed to target a virus known to infect CHO cells - cells that are commonly used to produce recombinant proteins and monoclonal antibodies - can potently block infection and viral replication. RNAi technologies to prevent or treat viral infection of manufacturing cell lines could provide a novel strategy to ensure product quality of biotherapeutic products for patients.
"By all accounts, these encouraging new findings are representative of the rapid progress we are making in this overall effort, and highlight important new applications of our biotherapeutics platform."
aWe remain excited about our Alnylam Biotherapeutics platform and how this application of RNAi technology has the potential to transform the manufacturing of biotherapeutic products. These new data describe applications of RNAi technologies to block viral infection of CHO cells - an approach that could preserve quality in biologics manufacturing and also maintain consistency of product supply,a said Ken Koblan, Ph.D., Chief Scientific Officer at Alnylam. aBy all accounts, these encouraging new findings are representative of the rapid progress we are making in this overall effort, and highlight important new applications of our biotherapeutics platform.a
In the new study, performed in collaboration with Dr. Ian Goodfellow from Imperial College London, siRNAs were designed and synthesized toward vesivirus, a virus known to infect CHO cells that can result in adverse effects on product quality and/or potential interruption in biologics manufacturing and product supply. Vesivirus is a small RNA virus in the calciviridae family that can be found in animal-derived products used to establish manufacturing cell banks and/or used during the course of manufacturing processes. siRNAs were designed toward the helicase and polymerase genes of vesivirus (strain 2117) and were screened for in vitro antiviral activity, resulting in identification of nine candidate siRNAs for further evaluation. In a treatment protocol, CHO cells grown in suspension culture were infected with vesivirus and then treated with anti-vesivirus siRNAs. The siRNA treatment was found to potently block viral replication with an approximate 2-log reduction in vesivirus RNA, as measured by PCR. In aggregate, these results provide key proof of concept for applications of RNAi technologies as an anti-viral strategy in biologics manufacturing that could also apply to other endogenous and exogenous viral cell culture pathogens.
About Alnylam Biotherapeutics
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 aa major scientific breakthrough that happens once every decade or so,a 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 Alnylama™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 worlda™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 Huntingtona™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 companya™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 majority 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 Alnylama™s future expectations, plans and prospects, including without limitation, Alnylama™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 companya™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 aRisk Factorsa 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 Alnylama™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.
