WALTHAM, Mass., Sept. 4 /PRNewswire-FirstCall/ -- 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 http://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 http://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 http://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 http://www.repligen.com.
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.
SOURCE Repligen Corporation
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Related links:
http://www.repligen.com
http://www.mda.org http://www.curefa.org
http://www.ataxia.org
CONTACT:
Walter C. Herlihy, Ph.D., President and Chief
Executive Officer, +1-781-419-1900, or Laura Whitehouse, VP,
Market Development, +1-781-419-1812, both of Repligen Corporation
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