Repligen receives $731,000 to support ongoing development of new treatments for Friedreich's ataxia

Dec 16 2009

Repligen Corporation (Nasdaq: RGEN) today announced that the Company has received $731,000 in research funding to support the ongoing development of new treatments for Friedreich's ataxia from the Muscular Dystrophy Association (MDA). This grant will support the completion of preclinical GLP toxicology testing and GMP manufacture of a drug candidate for human clinical trials. Support from the MDA not only provides important funding for Repligen's research but it also provides access to a global network of scientists, physicians and patients. This is the second research grant that Repligen has received from the MDA to support its Friedreich's ataxia program.

"We would like to thank the Muscular Dystrophy Association for their continued support of our Friedreich's ataxia program," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. "Funding from the MDA has allowed us to accelerate the development of a treatment that has the potential to be the first drug to modify the course of this very debilitating disease."

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. A potential clinical candidate synthesized by Repligen is being characterized in preclinical models to fully assess its pharmacologic, toxicologic and pharmacodynamic profile.