WALTHAM, Mass., Dec. 15 /PRNewswire-FirstCall/ -- Repligen Corporation 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.
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.
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 www.repligen.com.
Repligen Corporation
