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Repligen Receives Research Grants to Support Friedreich's Ataxia Development Program
Date:9/4/2008

pligen 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. Approx
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