In a breathtaking development, Alnylam and ISIS Pharmaceuticals announced tonight the formation of Regulus Therapeutics, a company solely focussed on the development of microRNA therapeutics. MicroRNAs are the endogenous counterparts of siRNAs used in RNAi Therapeutics and play key roles in gene regulation. Only 6 years after their discovery in humans, they have already been implicated in a number of diseases, and both microRNA inhibition and activation shows great promise in the treatment of cancer, viral and metabolic diseases and beyond.
Regulus Therapeutics, located in Carlsbad, California, near ISIS’ headquarters, combines Alnylam’s leading microRNA target IP estate (Tuschl III and siRNAs and microRNA mimics) with ISIS’ antisense and nucleic acid modification capabilities. This clearly creates the industry’s leading effort in microRNA-based drug development guided by a stunning line-up of leading scientists in microRNA research and gene regulation. As a pure play, Regulus Therapeutics should be ideally positioned to build on its existing assets in microRNAs. It further allows Alnylam management to concentrate on the development of RNAi Therapeutics while capitalising on their early IP in microRNAs.
Rosetta Genomics and Asuragen are the only two other notable microRNA-based companies left with ambitions both in diagnostics and therapeutics. It will be interesting how today’s announcement affects these two companies, particularly Rosetta’s existing significant relationship with ISIS. At the very least, it will help microRNAs get the attention of Wall Street and Big Pharma.
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