WALTHAM, Mass., April 11 /PRNewswire/ -- 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.
Friedreich's ataxia is an inherited neurodegenerative disease in which low
levels of the protein frataxin result in progressive damage to the nervous
system and loss of muscle function. Research in tissues derived from
patients as well as in mice indicates that the licensed compounds increase
production of the protein frataxin, 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.
Data supporting the ability of the licensed compounds to increase
production of the protein frataxin was published in Nature Chemical Biology
(August 20, 2006). This research was lead by Dr. Joel Gottesfeld, professor
of molecular biology at The Scripps Research Institute and supported in
part by the Friedreich's Ataxia Research Alliance (FARA). These compounds
are the only ones to date that have demonstrated utility in increasing both
the level of the frataxin protein in tissue samples from patients with
Friedreich's ataxia as well as frataxin gene activity in animal models.
Preliminary data from Dr. Gottesfeld's lab also suggests that these
compounds may have utility in treating other disorders such as Spinal
Muscular Atrophy and Huntington'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."
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.
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. The Scripps Research
Institute is headquartered in La Jolla, California.
About FARA
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. For more information, go to
http://www.curefa.org.
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 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.curefa.org
CONTACT:
Walter C. Herlihy, Ph.D., President and Chief
Executive Officer, +1-781-419-1900, or Laura Whitehouse, Vice
President, Market Development, +1-781-419-1812, both of Repligen
Corporation
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