Copernicus to Receive Nearly $1M From Cystic Fibrosis Foundation; Money to Support Clinical Trials of Non-Viral Gene Transfer in Subjects with Cystic Fibrosis.

Business Editors, Health & Medical Writers

CLEVELAND--(BW HealthWire)--March 12, 2002

Copernicus Therapeutics, Inc. to receive up to $937,000 from the Cystic Fibrosis Foundation to advance the development of therapeutic treatments for Cystic Fibrosis (CF). Copernicus' unique, non-viral approach to deliver the Cystic Fibrosis Transmembrane Regulator gene (CFTR) to the affected airway cells of CF patients may eventually provide a long-term treatment for this disease. To initiate the trials, the first subjects will receive Copernicus' non-viral gene therapy in the next few weeks.

"Our partnership with the Cystic Fibrosis Foundation reflects the critical need for an effective CF therapy and underscores the clinical potential of Copernicus' gene delivery and targeting technologies," said Robert C. Moen, M.D., Ph.D., President and Chief Executive Officer of Copernicus. "Copernicus' systemic approach is designed to bypass the difficulties encountered by other CF therapies. We expect that our PLASmin(TM) compaction technology will enable the delivery and efficient uptake of functional CFTR genes into the epithelial cells of the lung. PLASmin(TM) DNA complexes effectively deliver gene payloads without the immunological and inflammatory toxicities associated with viral vectors and competing non-viral vector systems"

"The CF Foundation is pleased to support the testing and development of new, safe, and effective therapeutics for people with cystic fibrosis," said Robert J. Beall, Ph.D., President and Chief Executive Officer of the CF Foundation. "We are encouraged by the progress made by Copernicus in their innovative approach to therapy. Gene-based therapeutics offer hope for a life-saving treatment that tackles the root cause of CF, rather than only treating the symptoms."

CF, a fatal genetic disease afflicting over 60,000 people in the United States and Europe, is caused by a defect in the gene expressing the CFTR protein. The disease produces a thick mucus that obstructs the airway, resulting in a heightened susceptibility to chronic infections and extensive lung damage which can lead to death in early adulthood.

Mark J. Cooper, M.D., Senior Vice President of Science and Medical Affairs for Copernicus said, "In recent years, medical research has explored new applications of DNA-based therapeutics for the treatment of CF. These drugs are designed to correct defective CFTR transport by providing a functional copy of the CFTR gene to epithelial cells lining a patient's airway passages and lungs. In order for such DNA-based drugs to have a therapeutic effect, however, these compounds must transport DNA into the nucleus of target cells, where it can achieve functional restoration of normal CFTR activity. Copernicus has developed proprietary methods to produce PLASmin(TM) Complexes, which are compacted DNA nanoparticles containing only a single nucleic acid molecule. PLASmin(TM) Complexes can much more efficiently cross the cell membrane and enter cell nuclei than DNA that has not been compacted. In addition, Copernicus has demonstrated that PLASmin(TM) Complexes produce high levels of nucleic acid expression in animal airway cells, can uniquely transfer DNA to non-dividing cells, resist physical and enzymatic destruction, and can be adapted for use with a wide range of nucleic acids. PLASmin(TM) Complexes are safe, non-immunogenic, and non-toxic in animals, and chronic administration in CF subjects may be possible without fear of inciting toxic inflammatory or immune responses."

The CF Foundation was established in 1955 to find a cure for CF and to improve the quality of life for the 30,000 children and young adults with the disease in the United States. Additional information can be accessed through the CF Foundation Web site at: http://www.cff.org. In 1997, the CF Foundation launched the Therapeutics Development Program to facilitate a pipeline of new therapies for CF. The program, in part, provides financial and early clinical support to biotechnology companies to stimulate the development of new therapeutics.

Copernicus Therapeutics, Inc., a privately held biotechnology company, is advancing novel targeting and delivery systems with broad applications in human therapeutics, DNA vaccines, and functional genomics. Copernicus' technologies include a targeting platform enabling the efficient uptake of drugs by specific cells and tissues, and a multi-component delivery platform that can be applied to nucleic acids to develop therapies for a variety of human diseases as well as to enhance transgene expression. The Company's targeting, delivery, and expression platforms are complementary and can be combined to enhance the efficacy and safety of existing drugs, to create novel therapeutics, and to speed up the drug discovery process. Additional information about Copernicus is available at http://www.cgsys.com.