Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office (USPTO) has issued Notices of Allowance for three patent applications from the company’s exclusively held Tuschl II patent estate. The USPTO has allowed claims in patent applications 12/537,602, 12/683,081 and 12/838,786 which cover compositions, methods, and uses of small interfering RNA (siRNA) that are important for the development and commercialization of RNAi therapeutics. These newly allowed claims are the first to emerge from the U.S. prosecution of the Tuschl II patent series that include composition of matter for siRNA, the molecules that mediate RNAi. The Tuschl II patent family is exclusively licensed to Alnylam for RNAi therapeutics on a worldwide basis through an agreement with Max Planck Innovation GmbH, the licensing agent for the Max Planck Society.
“We are very pleased with continued advancement of our Tuschl II patent estate through the USPTO, which further extends Alnylam’s leadership on intellectual property for RNAi therapeutics in the world’s largest pharmaceutical market,” said Laurence Reid, Ph.D., Senior Vice President, Chief Business Officer at Alnylam. “We believe that our unparalleled intellectual property estate will protect the innovative medicines we advance to the market as part of our ‘Alnylam 5x15’ product strategy. In the near term, Alnylam continues to realize value from this estate through our partnering efforts.”
“We are gratified that the USPTO has decided to allow these new composition of matter, method, and use claims from the Tuschl II patent estate,” said Dr. Joern Erselius, Managing Director, Max Planck Innovation GmbH. “The continued successful worldwide examination of the Tuschl II patent series highlights that this invention is seminal for RNAi, demonstrating the importance of the work performed by Professor Tuschl and other co-inventors at the Max Planck Institute.”
Patent applications 12/537,602, 12/683,081 and 12/838,786 include claims broadly covering compositions, methods, and uses for double-stranded RNAs having key structural elements that are widely recognized as important for the therapeutic activity of siRNA, including:
- two RNA strands with a length of 19-23 or 19-25 nucleotides;
- at least one strand forms a single-stranded 3’ overhang from 1-3 or 1-5 nucleotides;
- with or without chemical modifications; and
- the double-stranded RNA molecule cleaves a target mRNA at a single site.
The prosecution of these cases has been handled by the law firm of Lando & Anastasi, LLP.
Alnylam’s intellectual property position is comprised of fundamental, chemistry, delivery, and target patents and patent applications that the company believes are necessary for the development and commercialization of RNAi therapeutics. In aggregate, Alnylam owns or has in-licensed over 1,800 active patent cases, of which over 700 have issued or been granted worldwide, and over 300 have issued or been granted in the U.S., Europe, or Japan, the world’s largest pharmaceutical markets.
About RNA Interference (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 for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-AT3 for the treatment of hemophilia, ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-TMP for the treatment of hemoglobinopathies. As part of its “Alnylam 5x15TM” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in clinical development, including programs in advanced stages, on its own or with a partner by the end of 2015. Alnylam has additional partnered programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist, Ascletis, Monsanto, and Genzyme. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline, Sanofi, AstraZeneca and Biogen Idec. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for applications in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam’s VaxiRNA™ platform applies RNAi technology to improve the manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including, without limitation, statements regarding Alnylam’s expectations with respect to its “Alnylam 5x15” product strategy, and Alnylam’s views with regard to the strength, enforceability, and validity of its intellectual property estate, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Alnylam’s ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, as well as those risks more fully discussed in the “Risk Factors” section of its most recent quarterly report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.