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 http://www.repligen.com.
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