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Repligen Announces Publication of Positive Results with Proprietary HDAC Inhibitor in Huntington's Disease Model
Date:9/16/2008

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, 200
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