PipelineReview.com - Biotech News & Online Store - Alnylam Obtains Rights to RNAi Delivery Technology from The CBR Institute; Delivery Technology to Extend Alnylam's Leading Platform for RNAi Therapeutics

Alnylam Obtains Rights to RNAi Delivery Technology from The CBR Institute; Delivery Technology to Extend Alnylam's Leading Platform for RNAi Therapeutics

09 May 2006

CAMBRIDGE, MA, USA | May 09, 2006 | Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company,

announced today that it has obtained rights to RNAi delivery technology developed in the laboratory of Professor Judy Lieberman from the CBR Institute for Biomedical Research, a Harvard Medical School Affiliate. The in-licensed technology may enable small interfering RNAs - or siRNAs, the molecules that mediate RNAi - to be targeted with an engineered monoclonal antibody to tumors and other cell types, thereby broadening the scope of Alnylam's delivery technologies for RNAi therapeutics. The in-licensed technology, which was highlighted in an article published in the journal Nature Biotechnology in 2005, represents another potential approach for delivery of RNAi therapeutics with systemic administration.

"Our work in the area of RNAi demonstrates that antibody fusion proteins could potentially mediate both systemic and cell-type-specific delivery of siRNAs," said Judy Lieberman, M.D., Ph.D., Senior Investigator, CBR Institute for Biomedical Research and Professor of Pediatrics. "I am very excited to be collaborating with the scientific team at Alnylam, who are leaders in the field of RNAi and in the translation of RNAi science into products."

"Dr. Lieberman is a world-leading academic researcher in the field of RNAi technology for potential therapeutic applications. In-licensing this promising approach is part of our overall strategy to build the broadest platform of technology and intellectual property for the development of RNAi therapeutics," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam. "We believe our internal efforts, combined with access to technology from delivery-platform companies and premier academic labs such as Dr. Lieberman's, will continue to provide Alnylam with the leading capabilities in the field of RNAi."

In addition to this license agreement with CBR Institute, Alnylam recently entered into two relationships providing access to technologies for the systemic delivery of RNAi therapeutics. The first agreement was with Inex Pharmaceuticals Corporation and provides access to fundamental liposome delivery technology and intellectual property for systemic RNAi therapeutics. The second agreement was with the Massachusetts Institute of Technology (M.I.T.), and provides access to novel liposome technology being developed in the laboratory of Professor Robert S. Langer. Alnylam also recently announced the publication in Nature of the first demonstration of therapeutic gene silencing in non-human primates with systemic administration of siRNAs using liposomal delivery technology. Alnylam's expanding delivery toolkit for systemic delivery of siRNAs through the bloodstream is designed to make it possible to target disease-causing genes in a wide variety of tissues, and to increase the already broad potential of RNAi therapeutics to treat human disease.

About RNA Interference (RNAi)

RNA interference, or RNAi, is a naturally occurring mechanism within cells for selectively silencing and regulating specific genes. Since many diseases are caused by the inappropriate activity of specific genes, the ability to silence genes selectively through RNAi could provide a new way to treat a wide range of human diseases. RNAi is induced by small, double-stranded RNA molecules. RNAi is activated by chemically synthesized small interfering RNAs, or siRNAs, which are double-stranded RNAs that are targeted to a specific disease-associated gene. The siRNA molecules are used by the natural RNAi machinery in cells to cause highly targeted gene silencing. Alnylam's initial drug development programs are focused on Direct RNAi(TM) therapeutics which are siRNAs administered directly to diseased parts of the body. In parallel, the company is developing Systemic RNAi(TM) therapeutics that travel through the bloodstream to reach diseased parts of the body.

About Alnylam

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 building a pipeline of RNAi therapeutics; its lead program is in Phase I human clinical trials for the treatment of respiratory syncytial virus (RSV) infection, which is the leading cause of hospitalization in infants in the U.S. 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 Merck, Medtronic, and Novartis. The company, founded in 2002, maintains global headquarters in Cambridge, Massachusetts, and has an additional operating unit in Kulmbach, Germany. Alnylam is honored to be the "emerging/mid-cap" company recipient of the 2006 James D. Watson Helix Award, the biotechnology industry's award for outstanding corporate achievement. For more information, please visit www.alnylam.com.

About The CBR Institute of Biomedical Research

The CBR Institute for Biomedical Research is a non-profit, independent research institution located in Boston and recognized worldwide for discoveries that increase the body's ability to fight disease and to heal. The breakthroughs of CBRI scientists are greatly increasing our understanding of the influence of immune defense, inflammation, and genetics on biomedical discovery, healthcare, and disease management. The Institute's scientists, including three members of the National Academy of Science, are among the nation's leading per-capita recipients of funding from the National Institutes of Health. Collectively, they rank among the most cited immunology researchers in the world and their discoveries are being licensed to leading biotechnology and biopharmaceutical companies in the United States and abroad. The Institute is academically affiliated with Harvard Medical School, and its senior researchers hold faculty appointments in departments of the medical school.

For additional information, please visit www.cbrinstitute.org

Alnylam Forward-Looking Statements

Various statements in this release concerning our future expectations, plans, and prospects, including our views with respect to the development of RNAi therapeutics, 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 risks related to: our approach to discover and develop novel drugs, which is unproven and may never lead to marketable products; our ability to successfully incorporate and further develop in-licensed technology; obtaining, maintaining and protecting intellectual property utilized by our products; our ability to enforce our patents against infringers and to defend our patent portfolio against challenges from third parties; our ability to obtain additional funding to support our business activities; our dependence on third parties for development, manufacture, marketing, sales and distribution of our products; the successful development of products, all of which are in early stages of development; obtaining regulatory approval for products; competition from others using technology similar to ours and others developing products for similar uses; our dependence on collaborators; and our short operating history; as well as those risks more fully discussed in the "Risk Factors" section of our most recent report on Form 10-K on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We do not assume any obligation to update any forward-looking statements.

SOURCE: Alnylam Pharmaceuticals, Inc

Search in our News Channels

Search in our Online Store