Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced that it has secured key intellectual property (IP) for RNAi applications in vaccine development by completing an exclusive option agreement with Mount Sinai School of Medicine in New York City and an exclusive license agreement with The University of Queensland in Brisbane, Australia. These new applications of RNAi technology define opportunities for the advancement of novel vaccines in many human diseases, including infectious disease and cancer.

"RNAi technology has become a game changer across most of biomedical research, and its applications in the discovery and development of vaccine products are certainly no exception. We are pleased to have secured this intellectual property by obtaining key vaccine-related patents, which describe exciting opportunities for novel RNAi-based vaccine products in infectious disease and cancer," said Stuart Pollard, Ph.D., vice president, scientific and business strategy at Alnylam. "While our primary focus remains on the development of RNAi therapeutics, Alnylam's strategy is to also explore the full breadth of RNAi applications in medicine, such as our efforts on microRNA therapeutics with Regulus and our recent efforts with Alnylam Biotherapeutics. These new license agreements extend this strategy with emerging RNAi-based opportunities for vaccines."

Alnylam is collaborating with the laboratory of Benjamin tenOever, Ph.D. at the Mount Sinai School of Medicine on the development of microRNA-based attenuated influenza viruses which offer the potential for use as improved live attenuated influenza vaccines. In a paper published last year in Nature Biotechnology (Perez et al., Nature Biotechnology. 27:572-576, 2009), the tenOever lab demonstrated the ability to generate a live, attenuated influenza virus engineered with microRNA target sequences that provide attenuated replication in mammals without impaired growth characteristics during vaccine production. Specifically, microRNA-based attenuation of H1N1 and H5N1 influenza viruses resulted in novel viral antigens that conferred protection in mice from lethal flu infection. This technology is applicable to any virus amenable to recombinant production, and allows for the generation of viruses which can be efficiently propagated in one species and used as a live attenuated vaccine in another.

In addition, the company has exclusively licensed IP and technology from The University of Queensland around the use of RNAi in the development of cancer vaccines. The agreement was facilitated by the University's main commercialization company UniQuest Pty Limited. The new technology relates to work described in a paper published in 2009 in Proceedings of the National Academy of Sciences (PNAS) (Gu et al., Proc. Natl. Acad. Sci. USA, 106(20): 8314-8319, 2009) from the laboratory of Nigel McMillan, Ph.D. at The University of Queensland. In this paper, RNAi was used to generate truncated target gene mRNA transcripts in cancer cells. The truncated transcripts were then translated into incomplete proteins and found to be highly effective at inducing a tumour-protective immune response. The ability of using RNAi to mediate an effective immune response against tumors could have broad applications in cancer therapy.

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.