CYSTIC FIBROSIS PATIENT RECEIVES FIRST-OF-ITS-KIND GENE TRANSFER
Cleveland, Ohio, April 3, 2002—Copernicus Therapeutics, Inc., University Hospitals of Cleveland (UHC), Case Western Reserve University (CWRU) School of Medicine, and Cystic Fibrosis Foundation Therapeutics Inc. (CFFTI), a nonprofit affiliate of the Cystic Fibrosis Foundation, today announced that the first person in a 12 patient study has received a first-of-its-kind non-viral gene transfer vector in an effort to develop gene therapy for cystic fibrosis (CF). This study, being led by Dr. Michael Konstan, aims to use gene transfer technologies to provide a normal copy of the CF gene to correct the underlying defect. The mutations of this gene are the root cause of the disease. This trial is the first step in a long process toward developing a more effective therapy for individuals with CF.
“The CF Foundation’s mission is to assure the development of the means to cure and control cystic fibrosis and to improve the quality of life for those with the disease,” said Robert J. Beall, Ph.D., president and chief executive officer of the Cystic Fibrosis Foundation. “Copernicus is helping us realize this mission by tackling the root cause of CF, rather than only treating the symptoms. We will continue to work closely with Copernicus and are eagerly anticipating the results of this gene transfer trial and moving forward with this approach.”...
The new gene transfer vector was produced in Cleveland by Copernicus Therapeutics, Inc. whose unique, non-viral approach to deliver the CF gene to affected airway cells of CF patients may eventually provide a long-term treatment for this disease. Funding for this work was obtained from equity investors in Copernicus, as well as an award from CFFTI.
“This trial is the first step and a key indicator of the remarkable potential of Copernicus’ gene delivery technologies,” said Robert C. Moen, M.D., Ph.D., president and chief executive officer of Copernicus. “Copernicus’ approach is designed to bypass the immunological and inflammatory difficulties encountered by other CF gene transfer approaches. Researchers expect that the PLASmin™ compaction technology will enable the delivery and efficient uptake of functional CF genes into epithelial cells and result in a correction of the underlying defect. The initiation of the trial also is an example of how exciting technologies discovered at local academic centers can be transferred to a local biotechnology company for development into a potentially very valuable medical treatment."
“In recent years, medical research has explored new applications of DNA-based therapeutics for the treatment of CF. For such DNA-based drugs to have a therapeutic effect, however, they must safely and effectively be transported into the nucleus of target cells,” said Mark J. Cooper, M.D., senior vice president of science and medical affairs, Copernicus Therapeutics. “Copernicus’ development of
proprietary methods to produce compacted DNA nanoparticles containing only a single nucleic acid molecule puts us at the forefront of using DNA as a pharmaceutical.”
These DNA nanoparticles 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 compacted DNA nanoparticles are effective in animal airway cells, can uniquely transfer DNA to non-dividing cells, can resist physical and enzymatic destruction, and can be adapted for use with a wide range of nucleic acids.
Importantly, these nanoparticles have been shown to be safe, non-immunogenic, and non-toxic in animals, and chronic administration in people with CF may be possible without inciting toxic inflammatory or immune responses...Additional information about Copernicus is available at cgsys.com. |
