Contact: Alexander Shtifman
Phone: 617-713-8989

The Immune Disease Institute is proud to announce a new technology licensing agreement between Alnylam Pharmaceuticals, Inc. a leading RNA interference therapeutics company, and the laboratory of Senior Investigator Judy Lieberman, M.D., Ph.D.

Alnylam obtained rights to RNA interference (RNAi) delivery technology, developed in the Lieberman lab, which may enable small interfering RNAs - or siRNAs, 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.

The technology couples the homing ability of antibodies and an avid RNA-binding protein to deploy the RNAs in the bloodstream, where they can locate and slip into their target cells. First author on the Nature Biotechnology article is Erwei Song, M.D., a researcher in the Lieberman lab who is now a professor at Sun Yat Sen Medical University in China.

"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 Dr. Lieberman. "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. Their announcement of the licensing agreement can be found at

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 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.

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.

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