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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and Sylentis, S.A., a Spanish bio-pharmaceutical company devoted to the research and development of new therapies based on RNAi, announced that Alnylam has granted Sylentis a non-exclusive option for a new target-specific InterfeRx™ license. This license would enable the discovery, development, and commercialization of a synthetic siRNA directed towards an undisclosed target for the treatment of glaucoma. Sylentis’ program is currently in a Phase I/II clinical trial. Upon Sylentis’ exercise of this option, Alnylam would receive upfront and milestone payments, as well as royalties on sales of products covered by the licensing agreement. Additional financial details were not disclosed. Sylentis is a wholly owned subsidiary of Grupo Zeltia.

“We are pleased to be granting Sylentis a new InterfeRx license, thereby enabling their efforts with access to Alnylam intellectual property which we believe is critical for the development and commercialization of RNAi therapeutics,” said Laurence Reid, Ph.D., Senior Vice President and Chief Business Officer of Alnylam. “The agreement with Sylentis represents continued progress with our InterfeRx program, an important part of our overall strategy to create value today by leveraging our intellectual property portfolio.”

Alnylam created the InterfeRx licensing program to grant licenses under its intellectual property to biotechnology and pharmaceutical companies wishing to pursue RNAi therapeutics against specific targets outside Alnylam's core strategic interests. To date, a total of six companies are InterfeRx licensees; the license grants cover fourteen active target programs including four in clinical development. In addition to Sylentis, Alnylam's InterfeRx licensees include Calando Pharmaceuticals, Inc., Tekmira Pharmaceuticals Corporation, Quark Pharmaceuticals, Inc., GeneCare Research Institute Co., Ltd., and, under an option agreement, Benitec Ltd.

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.

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-APC for the treatment of hemophilia.


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