September 4, 2008

Repligen Receives Research Grants to Support Friedreich's Ataxia Development Program

Download and print (PDF)

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

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

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

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

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding current or future financial performance and position, management's strategy, plans and objectives for future operations, plans and objectives for product development, plans and objectives for present and future clinical trials and results of such trials, plans and objectives for regulatory approval, litigation, intellectual property, product development, manufacturing plans and performance such as the anticipated growth in the monoclonal antibody market and our other target markets and projected growth in product sales, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: the success of current and future collaborative relationships, the market acceptance of our products, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of product revenues and profits, our ability to generate future revenues, our ability to raise additional capital to continue our drug development programs, the success of our clinical trials, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights for our products, the risk of litigation regarding our intellectual property rights, our limited sales and manufacturing capabilities, our dependence on third-party manufacturers and value added resellers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in Repligen's filings with the Securities and Exchange Commission. Repligen assumes no obligation to update any forward-looking information contained in this press release or with respect to the announcements described herein.

The legacy of Marie Schlau: literature to help cure Friedreich's Ataxia

If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.

The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.

Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!


Research projects currently being financed by BabelFAmily

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.



Go to top