New Ventures in RNAi

There are many new therapeutic frontiers for our RNA technology, including applications in vaccine development, stem cell biology, bio-processing, genomics and biologics. We will continue to "innovate within innovation" and pursue these new high-value opportunities.

We have already created a new company for microRNA therapeutics, Regulus Therapeutics. microRNAs are small RNAs that exist naturally within mammalian cells and are believed to regulate the expression of over one-third of all human genes in the genome. They have been implicated in cancer, metabolic disease, viral infection, and immune disease, and we are excited about the potential for microRNA therapeutics that address entire pathways of disease.

Alnylam's Ventures

Regulus Therapeutics Inc.

In 2007, Alnylam and Isis created Regulus, the leading microRNA therapeutics company. Isis and Alnylam scientists and collaborators were the first to discover microRNA antagonist strategies that work in vitro and in vivo in animal studies (Krutzfeldt et al. (2005) Nature 438, 685-689; Esau et al. (2006) Cell Metab., 3, 87-98). Isis and Alnylam have also created and consolidated key IP believed by the companies to be required for development and commercialization of microRNA therapeutics. The company is based in Carlsbad, California. For more information, visit www.regulusrx.com.

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.