deacetylase 3 (HDAC 3) as an important enzyme target for therapeutic intervention in Friedreich's ataxia. These research findings confirm the drug target of the HDAC inhibitors that Repligen is currently developing for the treatment of inherited neurodegenerative diseases such as Friedreich's ataxia. The study entitled "Chemical Probes Identify a Role for Histone Deacetylase 3 in Friedreich's Ataxia Gene Silencing" published today in the journal Chemistry & Biology(
volume 16, 980-989, September 25, 2009 ) was conducted in collaboration with scientists at The Scripps Research Institute.
"Prior research indicated that HDAC enzymes play an important role in silencing the gene implicated in Friedreich's ataxia," stated Walther C. Herlihy, President and Chief Executive Officer of Repligen Corporation. "Identification of the involvement of HDAC 3 is an important step in developing a specific drug for Friedreich's ataxia without the potential toxicities associated with broad-acting HDAC inhibitors. There are more than 15,000 patients worldwide with Friedreich's ataxia with no therapies available for treatment."
Friedreich's ataxia is an inherited neurodegenerative disease caused by a single gene defect that results in inadequate production of the protein frataxin. Low levels of frataxin lead to degeneration of both the nerves controlling muscle movements in the arms and legs and the nerve tissue in the spinal cord. Preclinical studies have shown that specific HDAC inhibitors increase production of the protein frataxin which may have the potential to arrest disease progression in patients with Friedreich's ataxia. Several potential clinical candidates synthesized by Repligen are completing characterization in preclinical models to identify the compound with the appropriate pharmacologic, toxicologic and pharmacodynamic profile for human clinical trials. Repligen licensed the exclusive rights to intellectual property covering HDAC inhibitors from the Scripps Research Institute in April 2007 and our research efforts have been partially funded with grants from the Muscular Dystrophy Association, the Friedreich's Ataxia Research Alliance and the National Ataxia Foundation.
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
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www.repligen.com
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