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Copernicus: Delivering the Promise of Nucleic Acids as Therapeutics Nucleic acid-based therapies are a natural progression from development of genetic engineering in the 70's and the Human Genome Project of the late 90's. These emerging therapies introduce nucleic acids into cells to correct or modify genetic information to restore normal functionality. This technology can also silence aberrant or disease-causing genetic sequences. In each of these cases, nucleic acids intervene at the primary control point for cellular activity, gene expression. Nucleic acid therapeutics potentially offer novel therapies that cannot be addressed using conventional small molecule drugs or proteins. Additionally, these therapies provide a high level of cellular targeting with improved specificity of action, which can lower toxicity risks. A whole new way of treating disease is possible.

As stated by Nobel Prize winner Inder Verma, the major hurdle impeding nucleic acid-based therapies is "delivery, delivery, delivery." Dr. Verma is talking about the ability to safely and efficiently deliver nucleic acids to the cells of interest. Most of the initial attempts at delivery have involved the use of engineered viruses. This was a logical path to follow as Nature had already designed viruses to deliver their own nucleic acid into cells. However, many shortcomings have been associated with the use of engineered viruses, both minor and major (including death and causing cancer). Alternatively, non-viral approaches have been attempted and were often found to be inefficient, especially in non-dividing cells, which include essentially most of the cells of the body. Non-viral systems were also transient in their effects and often included toxicity issues. Copernicus Therapeutics, Inc. ("CTI") has developed an innovative non-viral approach based on condensing nucleic acids into nanoparticles, which has been found to avoid the many pitfalls associated with both viral and previous non-viral approaches. This CTI technology provides a very attractive alternative to prior delivery systems. The Company's nucleic acid nanotechnology platform is validated in humans with the development of a first-in-class therapeutic product for the treatment of Cystic Fibrosis. Additionally, CTI's collaborative research supports the development of first-in-class therapies to treat other serious diseases that have few therapeutic options, including diseases causing blindness (macular degeneration, diabetic retinopathy, and retinitis pigmentosa), airborne viral diseases (influenza A, SARS, and avian flu), and diseases of the brain (Parkinson's disease, stroke and Alzheimer's). The Copernicus technology addresses the delivery issue and is poised to deliver the promise of nucleic acids as therapeutics.

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