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Regeneron Pharmaceuticals and Alnylam Pharmaceuticals have allied to develop RNA interference-based drugs for the central nervous system (CNS) and the eye, opening a new chapter in the development of this rapidly emerging therapeutic modality. Until recently, delivery constraints have meant that indications involving the liver were prioritized for RNAi agents. But after more than a decade researching how to deliver short interfering RNA (siRNA) molecules into the CNS, the latter route is now on the brink of clinical application (Table 1). Also edging towards the clinic is Dicerna Pharmaceuticals, who entered a large-scale licensing deal with Eli Lilly last October, focused on neurodegenerative disease, pain and cardiometabolic disease. “We’re optimistic that in this calendar year we may, in collaboration with Lilly, have our first clinical candidate for a CNS indication,” Dicerna president and CEO Douglas Fambrough says.