MDA Awards $1 Million to Repligen for
Drug Development in Friedreich's Ataxia
TUCSON, Ariz., Sept. 4, 2008 - The Muscular Dystrophy Association (MDA) announced today that it has awarded approximately $1 million to Repligen Corp. of Waltham, Mass., for development of new compounds to treat Friedreich's ataxia (FA).
The compounds under development are histone deacetylase (HDAC) inhibitors, a class of chemical compounds that change the way cells interpret genetic information, usually activating genes that would otherwise be silent. When genes are activated, they serve as instruction manuals for production of proteins.
In FA, a mutation in the gene for the frataxin protein results in greatly reduced levels of frataxin and severe neurologic and cardiac problems. Frataxin is required to regulate cellular iron levels, which can accumulate to toxic levels without it. HDAC inhibitors may activate silent frataxin genes and restore frataxin protein production and iron regulation.
"We're very pleased to be giving Repligen this award," said Sharon Hesterlee, MDA's Vice President for Translational Research. "It's a great example of what translational research is all about - moving basic science findings from academic laboratories into drug development settings."
About MDA
MDA is a voluntary health agency working to defeat more than 40 neuromuscular diseases through programs of worldwide research, comprehensive services, and far-reaching professional and public health education. Friederich's ataxia is one of the diseases covered by the Association. To find out more about MDA, call (800) 572-1717, or visit www.mda.org.
About Repligen
Repligen Corporation is a biopharmaceutical company focused on the development of therapeutics for neurological disorders. A number of drug development programs are currently being conducted to evaluate drug candidates in diseases such as bipolar disorder and Friedreich's ataxia. For more information about Repligen, visit www.repligen.com. |