Springfield, VA — Repligen Corporation (NASDAQ: RGEN) announced today that it has entered into an exclusive commercial license with The Scripps Research Institute for intellectual property covering compounds, which may have utility in treating Friedreich's ataxia. The Scripps research was funded in part by grants from the Friedreich's Ataxia Research Alliance (FARA).
Friedreich's ataxia is an inherited neurodegenerative disease that causes severe and progressive damage to both the nerve tissue in the spinal cord and muscle tissue, especially of the arms,legs and heart. Research in patient cells and mice indicates that a class of compounds called Histone Deacetylase (HDAC) inhibitors increase production of the frataxin protein, which suggests potential utility of these compounds in slowing or stopping progression of the disease. There is currently no treatment or cure for Friedreich's ataxia.
Symptoms of Friedreich's ataxia typically emerge between the ages of 5 and 15 and often progress to severe disability, incapacitation or loss of life in early adulthood. Friedreich's ataxia is caused by a single gene defect that results in limited but inadequate production of the protein frataxin. The protein frataxin is essential for the proper functioning of the mitochondria, the power plant of both neural and muscle cells. Low levels of frataxin lead to degeneration of both the "sensory" nerves in the spinal cord and muscle tissue in the arms, legs and heart.
Data supporting the ability of this class of HDAC inhibitors to increase production of the protein frataxin was published in Nature Chemical Biology (August 20, 2006 online). This research was led by Dr. Joel Gottesfeld, professor of molecular biology at The Scripps Research Institute and supported in part by FARA. The compounds developed by Dr. Gottesfeld are the first HDAC inhibitors to demonstrate utility in increasing the level of frataxin protein in preclinical models of Friedriech's ataxia. Preliminary data from Dr. Gottesfeld's lab also suggests that this class of HDAC inhibitors might have utility in treating other disorders such as spinal muscular atrophy and Huntingdon's disease.
"We are very enthusiastic about Dr. Gottesfeld's work and we believe that this approach has the potential to modify the course of this serious and debilitating disease," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. "We look forward to working with Dr. Gottesfeld and his collaborators as well as patient advocacy groups like FARA in the development of what we hope will be an important new treatment for Friedreich's ataxia."
"FARA is very excited about the potential utility of these compounds in the treatment of Friedreich's ataxia," stated Ronald J. Bartek, President of The Friedreich's Ataxia Research Alliance. "We believe that this approach holds great promise for a therapy that could slow or arrest this devastating disease."
About Friedreich's Ataxia
Approximately one in every 50,000 people in the United States has Friedreich's ataxia, which is caused by a genetic defect that prevents adequate production of the protein frataxin essential for proper functioning of mitochondria, the energy producers for cells. Specific symptoms, which typically appear first between the ages of 5 and 15, include loss of strength and coordination in hands, arms and legs, slurred speech, reduced vision and hearing, scoliosis, heart disease, and diabetes. Friedreich's ataxia usually leads to full-time wheelchair use by about the age of twenty and death in early adulthood. There is currently no treatment or cure for Friedreich's ataxia.
FARA is a national, public, non-profit, tax-exempt organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich's ataxia. FARA's mission is to slow, stop, and reverse the damage caused by this disorder. http://www.CureFA.org
About The Scripps Research Institute
The Scripps Research Institute is one of the world's largest independent, non-profit biomedical research organizations, at the forefront of basic biomedical science that seeks to comprehend the most fundamental processes of life. Scripps Research is internationally recognized for its discoveries in immunology, molecular and cellular biology, chemistry, neurosciences, autoimmune, cardiovascular, and infectious diseases, and synthetic vaccine development. Established in its current configuration in 1961, it employs approximately 3,000 scientists, postdoctoral fellows, scientific and other technicians, doctoral degree graduate students, and administrative and technical support personnel. Scripps Research is headquartered in La Jolla, California.
About Repligen Corporation
Repligen Corporation is a biopharmaceutical company focused on the development of novel therapeutics for diseases that affect the central nervous system. In addition, we currently market two commercial products, Protein A and SecreFlo®, 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 http://www.repligen.com
President, Friedreich's Ataxia Research Alliance
Director, Public and Media Relations, Friedreich's Ataxia Research Alliance