"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 estab
|SOURCE Repligen Corporation|
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