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 orphan diseases.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.