Alnylam Consolidates Intellectual Property For RNA Activation (RNAa), A New Biological Discovery For Activation Of Gene Expression
RNAa May Have Applications in Many Human Diseases, Including Certain Genetic Disorders and Cancer
Company Completes Exclusive License Agreements with University of Texas Southwestern Medical Center, University of California San Francisco, and the Salk Institute for Biological Studies
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that it has consolidated the key intellectual property (IP) for RNA activation (RNAa), creating a dominant IP position in this new area of biology. RNAa is an emerging biological discovery involving double-stranded RNAs that target promoter regions in chromosomal DNA resulting in transcriptional activation of genes. The transcriptional activation, or up-regulation, of genes results in an increase in mRNA and protein production. Accordingly, this technology may have applications in a range of human disorders such as certain genetic diseases and cancer where the aberrant low expression of certain proteins is known to occur. In order to consolidate IP in this field, Alnylam has completed exclusive license agreements with the leading academic institutions working in this area: the Corey lab at the University of Texas Southwestern Medical Center (UT Southwestern); the Li lab at the University of California San Francisco (UCSF); and the Gage lab at the Salk Institute for Biological Studies (Salk).
"Alnylam is committed to scientific and IP leadership across all areas of modern biology's RNA revolution, including our ongoing efforts in RNAi and microRNA therapeutics, and now RNAa," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "While there's more to understand in this emerging biology, RNAa defines a new application for double-stranded RNAs that could have the potential to create an entirely new therapeutic platform for Alnylam. Many human diseases are caused by the abnormally low expression of proteins, and RNAa could be used to treat these disorders through selective transcriptional gene activation. Obtaining exclusive access to these key RNAa patents positions Alnylam to lead in the translational research of this biology as it advances toward in vivo validation and provides a solid foundation for business execution, as we've demonstrated in the past with our RNAi and microRNA platforms."
"We're excited to be working with Alnylam, as they have demonstrated a commitment to scientific excellence in their publications and academic collaborations," said David Corey, Ph.D., Professor of Pharmacology and Biochemistry at UT Southwestern. "We look forward to collaborating on the emerging science of RNAa biology to further explore the breadth of applications for transcriptional gene activation with agRNAs."
RNAa is mediated by double-stranded RNAs, possibly including endogenous miRNAs that target the promoter regions of genes in chromosomal DNA. So-called "anti-gene RNAs," or agRNAs, appear to increase transcription by complementary base-pairing with corresponding promoter sequences which enhance recruitment of the cellular RNA polymerase II and therefore increase the target gene's expression. In in vitro studies, agRNAs can increase the levels of a target gene's mRNA by nearly 10-fold, resulting in an increased level of the target gene's protein. The work by the Corey lab at UT Southwestern for the activation of the progesterone receptor was published last year (Janowski et al., Nature Chemical Biology 3, 166-173 (2007)) and a potential mechanism for RNAa is highlighted in a more recent paper (Schwartz et al., Nature Structural & Molecular Biology, doi:10.1038 / nsmb.1444(2008)). In addition, studies on RNAa have been performed by the Dahiya and the Li labs at UCSF (Li et al., PNAS, 103, 17337-17342 (2006); Chen et al., Mol Cancer Ther 7, 698-703 (2008); and Place et al., PNAS, 105, 1608-1613 (2008)) with a range of genes including VEGF, E-cadherin, and p21, as well as by the Gage lab (Kuwabara et al., Cell, 116, 779-793 (2004)) at the Salk. While additional work is necessary to understand the breadth of this biology, its applications in vivo, and its potential as a pharmacologic strategy, activation of gene expression with RNAa could offer new approaches for innovative medicines.
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. RNAi therapeutics target the cause of diseases by potently silencing specific messenger RNAs (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 applying its therapeutic expertise in RNAi to address significant medical needs, many of which cannot effectively be addressed with small molecules or antibodies, the current major classes of drugs. Alnylam is leading the translation of RNAi as a new class of innovative medicines with peer-reviewed research efforts published in the world's top scientific journals including Nature, Nature Medicine, and Cell. The company is leveraging these capabilities to build a broad pipeline of RNAi therapeutics; its most advanced program is in Phase II human clinical trials for the treatment of respiratory syncytial virus (RSV) infection. In addition, the company is developing RNAi therapeutics for the treatment of a wide range of disease areas, including hypercholesterolemia, liver cancers, and Huntington's disease. The company's leadership position in fundamental patents, technology, and know-how relating to RNAi has enabled it to form major alliances with leading companies including Medtronic, Novartis, Biogen Idec, Roche, Takeda, and Kyowa Hakko Kogyo. To reflect its outlook for key scientific, clinical, and business initiatives, Alnylam has established "RNAi 2010" which includes the company's plan to significantly expand the scope of delivery solutions for RNAi therapeutics, have four or more programs in clinical development, and to form four or more new major business collaborations, all by the end of 2010. Alnylam is a joint owner of Regulus Therapeutics LLC, a joint venture focused on the discovery, development, and commercialization of microRNA therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, visit www.alnylam.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, including its views with respect to the potential of RNAa, 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 risks related to: Alnylam's approach to discover and develop novel drugs, which is unproven and may never lead to marketable products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to enforce its patents against infringers and to defend its patent portfolio against challenges from third parties; Alnylam's ability to obtain additional funding to support its business activities; Alnylam's ability to realize future milestones and royalties as well as co-development and co-commercialization opportunities; Alnylam's dependence on third parties for development, manufacture, marketing, sales and distribution of products; obtaining regulatory approval for products; competition from others using technology similar to Alnylam's and others developing products for similar uses; Alnylam's dependence on collaborators; and Alnylam's short operating history; 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.
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