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Alnylam Receives Notice of Allowance from United States Patent and Trademark Office (USPTO) for New Patent Broadly Covering Conjugate-Based Delivery of RNA Therapeutics

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– Allowed Claims of Manoharan ’478 Application Cover GalNAc-Conjugates Independent of Length, Sequence, and Disease Target 

July 22, 2014

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for claims in the Manoharan et al. patent application 13/693,478. The ’478 patent application includes newly allowed claims directed to compositions including those comprising a modified RNA agent linked to a biantennary or triantennary ligand. Specifically, the allowed application includes claims that broadly cover single-stranded or double-stranded, chemically modified RNA therapeutics conjugated with an N-acetylgalactosamine (GalNAc) ligand independent of length, sequence, or disease target. The allowance of this patent application represents a major advance in Alnylam’s intellectual property (IP) estate, since GalNAc-siRNA conjugates enable potent and durable silencing of liver-expressed disease genes with subcutaneous dose administration and a wide therapeutic index. Importantly, this delivery approach is a key component of the company’s execution on its “Alnylam 5x15” and broader genetic medicine product strategy.

“These newly allowed claims from the Manoharan ’478 patent recognize the pioneering work of Alnylam scientists in the advancement of conjugates as a delivery platform for RNA therapeutics. We are gratified that the USPTO has acknowledged these innovations by allowance of broad claims from this patent application,” said Laurence Reid, Ph.D., Senior Vice President and Chief Business Officer of Alnylam. “Our conjugate delivery platform – specifically utilizing GalNAc-siRNA conjugates – enables subcutaneous dosing of RNAi therapeutics with potent and durable effects and a wide therapeutic index. With our Enhanced Stabilization Chemistry (ESC) conjugates, we believe that once-monthly and possibly once-quarterly subcutaneous dosing regimens can be achieved in our clinical pipeline programs. Based on this progress, we believe that GalNAc-conjugates are now the industry-leading delivery approach for RNA therapeutics targeting liver-expressed genes, and this patent allowance will reinforce Alnylam’s central role in value creation based on therapeutics linked to this technology.”

The allowed claims of the Manoharan ’478 patent application, as well as other granted, Alnylam-owned or -licensed patents, are provided on the company’s website, and in aggregate broadly cover delivery of RNAi therapeutics, including those that employ GalNAc-siRNA conjugate technology; additional claims from this and other patent families are pending. In addition, Alnylam’s owned or licensed patents broadly cover siRNAs and their use in a wide range of lengths from 15 to 49 nucleotides, and chemical modifications with naturally or non-naturally occurring nucleotides, including, for example, acyclic nucleotides such as “unlocked nucleoside analogs.” Alnylam’s IP estate also includes patents that broadly cover siRNAs toward a wide range of disease targets.

Alnylam will be providing an update on advances in the delivery of RNAi therapeutics with ESC-GalNAc-siRNA conjugates in an RNAi Roundtable webinar to be held at 11:00 a.m. ET today, and can be accessed by clicking here.

About RNAi
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 RNA (siRNA), 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.

About Alnylam Pharmaceuticals
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 as genetic medicines, including programs as part of the company’s “Alnylam 5x15TM” product strategy. Alnylam’s genetic medicine programs are RNAi therapeutics directed toward genetically defined targets for the treatment of serious, life-threatening diseases with limited treatment options for patients and their caregivers. These include: patisiran (ALN-TTR02), an intravenously delivered RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR) in patients with familial amyloidotic polyneuropathy (FAP); ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting TTR for the treatment of ATTR in patients with TTR cardiac amyloidosis, including familial amyloidotic cardiomyopathy (FAC) and senile systemic amyloidosis (SSA); ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD); ALN-CC5, an RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases; ALN-AS1, an RNAi therapeutic targeting aminolevulinic acid synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP); ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia; ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease; ALN-TMP, an RNAi therapeutic targeting TMPRSS6 for the treatment of beta-thalassemia and iron-overload disorders; ALN-ANG, an RNAi therapeutic targeting angiopoietin-like 3 (ANGPTL3) for the treatment of genetic forms of mixed hyperlipidemia and severe hypertriglyceridemia; ALN-AC3, an RNAi therapeutic targeting apolipoprotein C-III (apoCIII) for the treatment of hypertriglyceridemia; and other programs yet to be disclosed. As part of its “Alnylam 5×15″ strategy, as updated in early 2014, the company expects to have six to seven genetic medicine product candidates in clinical development – including at least two programs in Phase 3 and five to six programs with human proof of concept – by the end of 2015. Alnylam is also developing ALN-HBV, an RNAi therapeutic targeting the hepatitis B virus (HBV) genome for the treatment of HBV infection. The company’s demonstrated commitment to RNAi therapeutics has enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist, GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and Genzyme, a Sanofi company. In March 2014, Alnylam acquired Sirna Therapeutics, a wholly owned subsidiary of Merck. In addition, Alnylam holds an equity position in Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 200 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, Cell, the New England Journal of Medicine, and The Lancet. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

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