OPKO Health Announces Grant from Penn Center for Orphan Disease Research and Therapy
OPKO Health, Inc. (NYSE Amex: OPK) announced today that it has been
awarded a $200,000 research grant from the University of Pennsylvania
Center for Orphan Disease Research and Therapy to develop a therapeutic
agent to treat Mucopolysaccharidoses Type I (MPS I). The disease, called
Hurler disease, Sheie syndrome, or Hurler-Sheie Syndrome, depending on
severity, affects many body systems and can lead to organ damage. It
occurs in one in 100,000 births, and is caused by a defect in the gene
coding for the lysosomal enzyme, α-L-iduronidase (IDUA). As a result of
this genetic mutation, cells of affected individuals are either unable
to produce IDUA or produce it in low amounts. This results in an
inability of lysosomes to effect the stepwise degradation of certain
glycosaminoglycans (GAGs), namely dermatan sulfate and heparan sulfate,
a process essential for normal growth and maintenance of tissues.
OPKO has a proprietary AntagoNAT technology platform which involves
designing specific oligonucleotide molecules to target a non-coding
Natural Antisense Transcript of a target gene resulting in up-regulation
of the targeted gene to increase production of a protein, such as IDUA.
Penn Center for Orphan Disease Research and Therapy
The Penn Center for Orphan Disease Research at the Raymond and Ruth
Perelman School of Medicine at the University of Pennsylvania is an
interdisciplinary center focused on discovering novel treatments for
About OPKO Health, Inc.
We are a multi-national biopharmaceutical and diagnostics company that
seeks to establish industry-leading positions in large and rapidly
growing medical markets by leveraging our discovery, development and
commercialization expertise and our novel and proprietary technologies.
Certain of the statements made in this press release are
"forward-looking statements," as that term is defined under the Private
Securities Litigation Reform Act of 1995 (PSLRA), which statements may
be identified by words such as "expects," "plans," "projects," "will,"
"may," "anticipates," "believes," "should," "could," "intends,"
"estimates," and other words of similar meaning, including statements
regarding the effectiveness of our technology platform, therapeutic
modality for up-regulation of the targeted gene to increase production
of a protein, and our ability to develop and commercialize therapeutic
agents to treat MPS1. Many factors, including those described in our
filings with the Securities and Exchange Commission, could cause actual
results or developments to differ materially from those projected or
implied in these forward-looking statements, including that clinical
trials may fail and not be successful or achieve the expected results or
effectiveness, and may not generate data that would support the approval
or marketing of these or other products, that others may develop
products, including other early stage products which are superior to the
products we are developing, and that the products, if developed, may not
have advantages over other marketed products. In addition,
forward-looking statements may also be adversely affected by risks
inherent in funding, developing and obtaining regulatory approvals of
new, commercially-viable and competitive products and treatments,
general market factors, competitive product development, product
availability, federal and state regulations and legislation, the
regulatory process for new products and indications, manufacturing
issues that may arise, patent positions and litigation, among other
factors. The forward-looking statements contained in this press release
speak only as of the date the statements were made, and we do not
undertake any obligation to update forward-looking statements. We intend
that all forward-looking statements be subject to the safe-harbor
provisions of the PSLRA.
OPKO Health, Inc.
Steven D. Rubin, 305-575-6015
Source: OPKO Health, Inc.
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