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Alnylam Provides Key 2013 – 2014 Goals For RNAi Therapeutics Pipeline

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today its key “Alnylam 5x15™” pipeline and partner program goals for 2013 through 2014.

“In 2012, we made tremendous progress in our RNAi therapeutic development efforts with our ‘Alnylam 5x15’ product strategy focused on genetically defined targets for diseases with limited treatment options for patients and their caregivers. The next couple of years promise to be transformative for Alnylam as we aim to advance our pipeline into Phase III, report on key clinical data in multiple programs, and expand the breadth our ‘5x15’ pipeline with additional clinical and pre-clinical programs,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “Specifically, we aim to advance what we believe to be the industry leading effort in ATTR, with ALN-TTR02 starting a Phase III trial for FAP patients and ALN-TTRsc entering a pilot Phase II trial in FAC patients. In addition, we expect to report on clinical results from Phase II and Phase I studies from these two programs respectively, in mid-2013. Further, we are expanding our ‘5x15’ clinical pipeline with ALN-AT3, a potential breakthrough therapy for hemophilia, including hemophilia complicated by ‘inhibitors,’ and for other rare bleeding disorders, where we expect to file an IND in mid-2013 and initiate a Phase I study in late 2013. Finally, we are excited today to introduce a new ‘Alnylam 5x15’ program – ALN-AS1 for the treatment of acute intermittent porphyria, an ultra-rare genetic disease caused by loss of function mutations in an enzyme in the heme biosynthesis pathway that result in acute and/or recurrent life-threatening attacks with severe abdominal pain, peripheral and autonomic neuropathy, and neuropsychiatric manifestations. In aggregate, we believe that execution on our ‘Alnylam 5x15’ programs and our ambitious goals for 2013 and beyond will enable continued advancement of RNAi therapeutics as innovative medicines for patients, resulting in value creation for our shareholders.”

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