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Alnylam Pharmaceuticals, Inc. and Regeneron Pharmaceuticals, Inc. have established a new RNAi collaboration for the development of ocular and CNS treatments

Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) and Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN), have announced a collaboration to “discover, develop and commercialize new RNA interference (RNAi) therapeutics for a broad range of diseases” focused on ocular and central nervous system (CNS) indications. It is understood that Regeneron will pay a $400 million upfront payment to Alnylam and will also purchase up to $400 million of Alnylam equity at $90 per share.  The companies will select an as yet undisclosed number of preclinical disease programs.


“Ribonucleic acid interference” (or “RNAi”), a biological process originally thought to have evolved to counter viral infections, is continually finding innovative applications for the treatment of a variety of human diseases.  There is a rather ironic connection between RNAi and the human eye in the story of how this phenomenon was first identified.  In an effort to make flowers more aesthetically pleasing to the eye with deeper petal colours, plant genetic researchers back in the 1990s were experimenting with genes known to be responsible for flower pigmentation.  By introducing such genes into plants, researchers expected to breed varieties with more vivid colours but instead got the opposite result, i.e., flowers with either variegated pigmentation or flowers lacking pigmentation altogether!  Introducing such genes, it appeared, somehow effected the normal expression of flowers’ endogenous pigment genes. Curiosity as to how such an effect was occurring led a number of researchers by the mid-1990s to discover that when plants were attacked by certain viruses one of the defence mechanisms used by the plant to rid itself of the pathogen involved a targeted destruction of viral RNA.  The rationale here was that by destroying the virus’ ability to translate viral RNA into viral protein, the plant could halt the infection in its tracks.  The phenomenon was termed “PTGS” or “post transcriptional gene silencing” but is today commonly referred to as RNA interference (RNAi).  RNAi is essentially a biological mechanism that interferes with the information flow between RNA and protein and this has built up to a 2-decade biotechnology industry which followed up to the recent commercial agreement between Alnylam and Regeneron.


According to the announcement, Alnylam stated that, “[u]nder the terms of the alliance, Alnylam will work exclusively with Regeneron to discover RNAi therapeutics for eye and CNS diseases. Regeneron will lead development and commercialization for all programs targeting eye diseases, with Alnylam entitled to potential milestone and royalty payments. The companies will jointly advance and alternate leadership on CNS programs, with the lead party retaining global development and commercial responsibility. For CNS programs, both companies will have the option at candidate selection to participate equally in potential future profits of programs led by the other party”.