Repligen Announces Identification Of Drug Target For Friedreich's Ataxia

Main Category: Neurology / Neuroscience
Also Included In: Genetics;  Biology / Biochemistry
Article Date: 26 Sep 2009 - 2:00 PDT

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Repligen Corporation (Nasdaq: RGEN) reported publication of research that identifies histone 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.

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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