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September 4, 2008
Repligen Receives Research Grants to Support Friedreich's Ataxia Development Program
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WALTHAM, MA - September 4, 2008 - Repligen Corporation (NASDAQ: RGEN) today announced that the Company has received $1.125 million in research grants to support the development of new treatments for Friedreich's ataxia. The grants are comprised of $1 million from the Muscular Dystrophy Association (MDA) and $125,000 from the Friedreich's Ataxia Research Foundation (FARA) and the National Ataxia Foundation (NAF). The grants will further the development, characterization and selection of a drug candidate for human clinical trials as well as support the development of tools ("biomarkers") to monitor the desired biological impact of the drugs in clinical trials.
"We are very pleased to receive support from the Muscular Dystrophy Association, the Friedreich's Ataxia Research Foundation and the National Ataxia Foundation, national organizations recognized for their commitment to innovative research aimed at providing new therapeutic options for patients," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. "We believe that establishing partnerships with these non-profit organizations will result in a number of mutual benefits as the program matures and we prepare for human clinical trials."
Friedreich's ataxia is an inherited neurodegenerative disease in which a single gene defect results in inadequate production of the protein frataxin, leading to progressive damage to the nervous system and loss of muscle function. Prior research has demonstrated that production of frataxin protein can be increased in tissue samples and animal models by a family of compounds, suggesting the potential utility of these compounds in slowing or halting disease progression.
In April 2007, Repligen established a development program for Friedreich's ataxia. Over the past year, the Company has made significant progress in advancing this program, resulting in the identification of advanced lead compounds with improved potency and specificity. These lead compounds are currently being assessed in pharmacology and toxicology in order to determine if one is suitable for clinical development in Friedreich's ataxia. Repligen is also evaluating this family of compounds for activity in preclinical models of other neurodegenerative diseases including Huntington's disease and spinal muscular atrophy. The MDA grant will partially support the characterization of the lead compounds, including the evaluation of compounds in preclinical models of disease and the completion of the pharmacology and toxicology. The FARA and NAF grant will support the development of new methods to quantitate the impact of drug treatment on frataxin gene expression and frataxin protein in preclinical models and in patients. In further support of this development program, Repligen previously announced receipt of a grant from Go FAR (Friedreich's Ataxia Research), an Italian-based fundraising organization dedicated to raising donations for Friedreich's ataxia research, to develop a biomarker, which may be useful in monitoring the biochemical activity and guiding the dosing of a clinical candidate in patients.
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 inadequate production of the protein frataxin. The protein frataxin appears to be 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 nerves controlling muscle movements in the arms and legs and the nerve tissue in the spinal cord. Approximately one in every 50,000 people in the United States has Friedreich's ataxia. There is currently no treatment or cure for Friedreich's ataxia.
About the Muscular Dystrophy Association
The Muscular Dystrophy Association is a voluntary health agency working to defeat more than 40 diseases through programs of worldwide research, comprehensive services, and far-reaching professional and public health education. Friedreich's ataxia is one of the diseases covered by MDA. For more information, go to www.mda.org.
About the Friedreich's Ataxia Research Foundation
The Friedreich's Ataxia Research Foundation 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. For more information, go to www.curefa.org.
About the National Ataxia Foundation
The National Ataxia Foundation is a membership supported, non-profit organization established in 1957 to help persons with ataxia and their families. The Foundation's primary purpose is to support promising ataxia research and to provide vital programs and services for ataxia families. For more information, go to www.ataxia.org.
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.
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Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:
1) Gene Therapy for Friedreich's Ataxia research project:
The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.
2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.
The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.