Cleveland-based biotechnology firm Copernicus Therapeutics Inc. produced the non-viral gene transfer system used in the clinical trial. Working together with UHC and CWRU researchers, Copernicus formulated a way to "compact" or tightly bind strands of DNA so that it is tiny enough to pass through a cell membrane and into the nucleus. The ultimate goal is for the DNA to produce a protein needed by people with CF to correct the basic defect in CF cells.
"The primary goal of this Phase I study was to determine if this gene therapy method is safe and tolerable as administered in this trial. All participants in this study completed the trial without significant side effects and the treatment itself was well-tolerated," stated Michael W. Konstan, M.D., Associate Professor of Pediatrics at CWRU and Director of the LeRoy Matthews Cystic Fibrosis Center, Rainbow Babies & Children's Hospital of UHC. "The secondary goals of the trial were to evaluate if the CF gene was successfully transferred to airway cells and if it functioned normally, results which would suggest that this therapy may be of benefit to people with CF. Our data were very encouraging with indications that this gene transfer may have occurred."
"This gene therapy research has exciting potential as a new approach to addressing the genetic root cause of CF," said Robert J. Beall, Ph.D., president and chief executive officer
Contact: Eileen Korey
University Hospitals of Cleveland