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Press release from GlobeNewswire (a Nasdaq OMX company)

Athersys Receives Orphan Drug Designation for MultiStem in Hurler's Syndrome

Tuesday, July 10, 2012

Athersys Receives Orphan Drug Designation for MultiStem in Hurler's Syndrome08:00 EDT Tuesday, July 10, 2012CLEVELAND, July 10, 2012 (GLOBE NEWSWIRE) -- Athersys, Inc. (Nasdaq:ATHX) announced today that MultiStem®, Athersys' proprietary cell therapy, has been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of Hurler's Syndrome, also known as mucopolysaccharidosis type I or MPS-I. Hurler's Syndrome is a lysosomal storage disorder that affects approximately 1 of every 100,000 children. Orphan drug designation, which is intended to facilitate drug development, provides substantial potential benefits to the sponsor, including funding for certain clinical studies, study-design assistance, tax incentives and seven years of market exclusivity for the product upon regulatory approval. Lysosomal storage diseases are a group of rare, inherited diseases that initially result from defects in the lysosome, a component of the cell that is responsible for the breakdown of certain molecules, such as complex lipids and sugars. In such cases, the lysosome produces too little of an enzyme that is necessary for the break-down of these materials, resulting in unwanted accumulations in the cells and tissues, which can lead to neurological disease and a range other complications. Hurler's Syndrome is caused by a deficiency of a specific enzyme responsible for degrading complex sugars called glycosaminoglycans (GAGs), accumulation of which can lead to mental retardation, respiratory and cardiac complications, and death in early childhood. In a recent publication in the scientific journal, Cell Transplantation, investigators at the University of Minnesota, together with scientists from Athersys, described preclinical study results suggesting that MultiStem cells could provide benefit to patients suffering from lysosomal storage disorders, such as Hurler's Syndrome. The study demonstrated that an injection of MultiStem cells in neonatal MPS-I mice reduces the accumulation of GAGs in the brain thereby resulting in improvements in behavioral and motor function in the diseased mice. The company also recently announced the issuance of a U.S. patent covering the use of non-embryonic, multipotent stem cells, such as MultiStem, for the treatment of lysosomal storage diseasesAbout MultiStem MultiStem® cell therapy is a patented product that has shown the ability to promote tissue repair and healing in a variety of ways, such as through the production of multiple therapeutic factors produced in response to signals of inflammation and tissue damage. MultiStem has demonstrated therapeutic potential for the treatment of inflammatory and immune disorders, neurological conditions, and cardiovascular disease, as well as other areas, and represents a unique "off-the-shelf" stem cell product that can be manufactured in a scalable manner, may be stored for years in frozen form, and is administered without tissue matching or the need for immune suppression. The product is extensively characterized for safety, consistency and potency. Athersys has forged strategic partnerships with Pfizer Inc. to develop MultiStem for inflammatory bowel disease and with RTI Biologics, Inc. to develop cell therapy for use with a bone allograft product in the orthopedic market.About Athersys Athersys is a clinical stage biotechnology company engaged in the discovery and development of therapeutic product candidates designed to extend and enhance the quality of human life. The Company is developing its MultiStem® cell therapy product, a patented, adult-derived "off-the-shelf" stem cell product platform for disease indications in the cardiovascular, neurological, inflammatory and immune disease areas. The Company currently has several clinical stage programs involving MultiStem, including for treating inflammatory bowel disease, ischemic stroke, damage caused by myocardial infarction, and for the prevention of graft versus host disease. Athersys has also developed a diverse portfolio that includes other technologies and product development opportunities, and has forged strategic partnerships and collaborations with leading pharmaceutical and biotechnology companies, as well as world-renowned research institutions in the United States and Europe to further develop its platform and products. More information is available at www.athersys.com. The Athersys, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=4548.Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties. These forward-looking statements relate to, among other things, the expected timetable for development of our product candidates, our growth strategy, and our future financial performance, including our operations, economic performance, financial condition, prospects, and other future events. We have attempted to identify forward-looking statements by using such words as "anticipates," "believes," "can," "continue," "could," "estimates," "expects," "intends," "may," "plans," "potential," "should," "suggest," "will," or other similar expressions. These forward-looking statements are only predictions and are largely based on our current expectations. A number of known and unknown risks, uncertainties, and other factors could affect the accuracy of these statements. Some of the more significant known risks that we face that could cause actual results to differ materially from those implied by forward-looking statements are the risks and uncertainties inherent in the process of discovering, developing, and commercializing products that are safe and effective for use as human therapeutics, such as the uncertainty regarding market acceptance of our product candidates and our ability to generate revenues, including MultiStem for the treatment of inflammatory bowel disease, acute myocardial infarction, stroke and other disease indications, including lysosomal storage disorders, and the prevention of graft-versus-host disease. These risks may cause our actual results, levels of activity, performance, or achievements to differ materially from any future results, levels of activity, performance, or achievements expressed or implied by these forward-looking statements. Other important factors to consider in evaluating our forward-looking statements include: our ability to raise additional capital; final results from our MultiStem clinical trials; the possibility of delays in, adverse results of, and excessive costs of the development process; our ability to successfully initiate and complete clinical trials; changes in external market factors; changes in our industry's overall performance; changes in our business strategy; our ability to protect our intellectual property portfolio; our possible inability to realize commercially valuable discoveries in our collaborations with pharmaceutical and other biotechnology companies; our ability to meet milestones under our collaboration agreements; our collaborators' ability to continue to fulfill their obligations under the terms of our collaboration agreements; our possible inability to execute our strategy due to changes in our industry or the economy generally; changes in productivity and reliability of suppliers; and the success of our competitors and the emergence of new competitors. You should not place undue reliance on forward-looking statements contained in this press release, and we undertake no obligation to publicly update forward-looking statements, whether as a result of new information, future events or otherwise. CONTACT: William (B.J.) Lehmann, J.D. President and Chief Operating Officer Tel: (216) 431-9900 bjlehmann@athersys.com Investor Relations: Lisa M. Wilson In-Site Communications Tel: (917) 543-9932 lwilson@insitecony.com