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Repligen Announces Publication of Positive Results with Proprietary HDAC Inhibitor in Huntington's Disease Model

Study Published in the Proceedings of the National Academy of Sciences

WALTHAM, Mass., Sept. 16 /PRNewswire-FirstCall/ -- Repligen Corporation (Nasdaq: RGEN) today reported publication of a preclinical study demonstrating that a novel histone deacetylase (HDAC) inhibitor improved disease symptoms in a transgenic animal model of Huntington's disease. The study, led by scientists at The Scripps Research Institute, demonstrated that oral administration of the drug candidate to the mice after the onset of symptoms slowed the progression of disease. Treated animals showed superior motor performance by multiple measures, reduced loss of body weight, reduced brain atrophy and improved overall appearance compared to untreated animals. Huntington's mice were also analyzed for changes in the hundreds of genes whose expression in the brain is altered in the mouse model of Huntington's, as well as in humans with Huntington's disease. Using gene microarrays, the researchers identified genes in three brain regions whose expression was altered in the Huntington's mice and whose expression was altered by treatment with the HDAC inhibitor. Treatment partially normalized the expression level of approximately 90% of these genes with 32% being restored to normal levels. These results suggest that an HDAC inhibitor may be useful in treating Huntington's disease and that specific genes may be useful as biomarkers in clinical trials. The research was conducted using a compound that is covered by Repligen's exclusive license from The Scripps Research Institute. The study entitled "The HDAC Inhibitor 4b Ameliorates the Disease Phenotype and Transcriptional Abnormalities in Huntington's Disease Transgenic Mice" will be published the week of September 15, 2008 in the online version of the Proceedings of the National Academy of Sciences.

"The marked reduction in symptoms achieved in the Huntington's disease model without overt toxicity defines a second disease target for our HDAC inhibitor program," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. "We plan to continue to evaluate the utility of our novel HDAC inhibitors as a potential treatment for both Friedreich's ataxia and Huntington's disease."

Huntington's disease is caused by a trinucleotide repeat expansion in the Huntington's disease gene (Htt) that results in production of a mutant misfolded protein that is unable to function correctly. Huntington's disease is characterized by dysregulation in the transcription of hundreds of genes in the brain, leading to Huntington's symptoms ranging from jerky and random movements to impaired thinking and perception. Huntington's disease is a familial disease, passed from parent to child through a mutation in the normal gene. Symptoms of Huntington's disease typically emerge between the ages of 30 and 50 and fall into three categories: motor, cognitive and psychiatric. Cognitive symptoms include slowed processing of information in the brain, resulting in communication and planning difficulties, while depression is the most common psychiatric symptom of Huntington's disease. Motor symptoms include lack of coordination, muscle spasms, and chorea. As the disease progresses, any function requiring muscle control is affected, leading to severe disability, incapacitation or loss of life due to complications 10 to 20 years after symptoms first appear. There are approximately 30,000 people in the United States with Huntington's disease and there is currently no safe and effective treatment for the disease.

Repligen licensed the exclusive rights to intellectual property covering HDAC inhibitors from The Scripps Research Institute in April 2007 following which Repligen established a HDAC development program for Friedreich's ataxia. Over the past year, the Company has made significant progress in advancing this program, resulting in the identification of advanced lead compounds with improved potency and specificity. These lead compounds are currently being assessed in pharmacology and toxicology models in order to determine if one is suitable for clinical development. In addition to Huntington's disease, Repligen is evaluating this family of compounds for activity in preclinical models of other neurodegenerative diseases including spinal muscular atrophy.

About The Scripps Research Institute

The Scripps Research Institute is one of the world's largest independent, non-profit biomedical research organizations, at the forefront of basic biomedical science that seeks to comprehend the most fundamental processes of life. Established in its current configuration in 1961, it employs approximately 3,000 scientists, postdoctoral fellows, scientific and other technicians, doctoral degree graduate students, and administrative and technical support personnel and is headquartered in La Jolla, California.

About Repligen Corporation

Repligen Corporation is a biopharmaceutical company focused on the development of novel therapeutics for neurological disorders. In addition, we are the world's leading supplier of recombinant Protein A, the sales of which partially fund the advancement of our development pipeline while supporting our financial stability. Repligen's corporate headquarters are located at 41 Seyon Street, Building #1, Suite 100, Waltham, MA 02453. Additional information may be requested from

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding current or future financial performance and position, management's strategy, plans and objectives for future operations, plans and objectives for product development, plans and objectives for present and future clinical trials and results of such trials, plans and objectives for regulatory approval, litigation, intellectual property, product development, manufacturing plans and performance such as the anticipated growth in the monoclonal antibody market and our other target markets and projected growth in product sales, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: the success of current and future collaborative relationships, the market acceptance of our products, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of product revenues and profits, our ability to generate future revenues, our ability to raise additional capital to continue our drug development programs, the success of our clinical trials, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights for our products, the risk of litigation regarding our intellectual property rights, our limited sales and manufacturing capabilities, our dependence on third-party manufacturers and value added resellers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in Repligen's filings with the Securities and Exchange Commission. Repligen assumes no obligation to update any forward-looking information contained in this press release or with respect to the announcements described herein.

SOURCE Repligen Corporation
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