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Extended RNAi delivery deal
CAMBRIDGE, Mass.—In an agreement that Alnylam Pharmaceuticals COO Barry Greene describes as "focused on delivery of RNAi from outside to inside cells," the company has announced a five-year extension of its research program at the Massachusetts Institute of Technology Center for Cancer Research. Robert Langer, an Institute Professor at MIT who also sits on Alnylam's scientific advisory board, and Daniel Anderson, in the MIT Center for Cancer Research, are the co-investigators of the research program. Alnylam will provide research funding for ten post-doctoral researchers annually over the five-year term. Other financial terms of the agreement are not being disclosed. The agreement provides Alnylam an exclusive option to license future RNAi technology resulting from the research sponsorship.
Green suggests that RNAi "is the kind of fundamental breakthrough that only happens about once a decade," and notes that it provides a different therapeutic pathway than small molecules and monoclonal antibodies, which are now the two major classes of drugs. This pathway provides a mechanism for controlling the flow of genetic information. RNA interference is activated when RNA molecules occur as double-stranded pairs in the cell. This RNA activates biochemical machinery which degrades those mRNA molecules that carry a genetic code identical to that of the double-stranded RNA. When such mRNA molecules disappear, the corresponding gene is silenced and no protein of the encoded type is made.
Alnylam—named for the middle star in Orion's belt—was formed in 2002 to exploit RNAi. Within three years, Novartis AG came on board when Alnylam and the Novartis Institute for BioMedical Research (NIBR) formed an alliance valued at more than $700 million to discover, develop and commercialize RNAi therapeutics across multiple therapeutic areas. In February 2006, Alnylam and Novartis formed a second collaboration to jointly advance RNAi therapeutics for pandemic flu.
"We have made important advancements already this year, and anticipate continued progress on many fronts in the months to come," says John Maraganore, Ph.D., company president and CEO. "Our lead program, ALN-RSV01, an RNAi therapeutic for the treatment of RSV infection, is advancing towards Phase II studies that hold the potential of demonstrating human proof of concept as measured by anti-viral activity."
Respiratory syncytial virus (RSV) infects nearly every child at least once by the age of two and accounts for more than 125,000 hospitalizations annually in the U.S. pediatric population, the company claims, and also poses a great risk to the elderly and other adults with compromised immune systems.
Alnylam has a development program underway for the treatment of liver cancers and potentially other solid tumors, Barry Greene adds. The company is using "lipidoid-based technology" that was developed in the Langer laboratory to engineer the drug to a specific particle size, protect it in the bloodstream and use a cationic charge to ensure uptake by the liver.
Alnylam is also collaborating with Medtronic on the development of novel drug-device combinations incorporating RNAi therapeutics to treat neurodegenerative disorders such as Parkinson's, Huntington's and Alzheimer's. Initial work is focused on delivering RNA therapeutics to specific locations within the brain via novel infusion systems. Under the terms of the agreement, Alnylam will be responsible for discovery and early clinical development of the RNAi therapeutic products and Medtronic will be responsible for late-stage development and commercialization of the drug-device product