Copernicus Announces Breakthrough in Non-Viral Gene Therapy

CLEVELAND--(BUSINESS WIRE)--Sept. 23, 2003--Scientists at Copernicus Therapeutics, Inc. and Case Western Reserve University have published a manuscript in the Journal of Biological Chemistry showing that compacted DNA nanoparticles can effectively transfect non-dividing human cells. This achievement removes a major barrier to effective non-viral gene therapy, permitting highly efficient gene transfer results following direct in vivo dosing of animals and humans.

Mark J. Cooper, M.D., Copernicus' Senior Vice President of Science and Medical Affairs said, "This manuscript demonstrates that our formulation of compacted DNA nanoparticles consists of single molecules of DNA which are condensed to their minimum possible volume using positively charged peptides. We have long speculated that the small size of DNA nanoparticles might account for their unique ability to productively express DNA in differentiated human cells. We now show that this gene transfer characteristic of DNA nanoparticles is directly related to its diameter, with enhanced efficiency when the particle size is less than the diameter of the nuclear membrane pore. Since the vast majority of human cells have intact nuclear membranes during the time interval of DNA transfer, the ability to efficiently transfect non-dividing human cells is a necessary property for an effective non-viral gene transfer technology. These experimental findings provide insights into one of the possible mechanisms that may account for our extraordinarily high gene transfer results in the airways of mice and the nasal epithelium of cystic fibrosis (CF) subjects."

These studies were conducted in collaboration with Drs. Richard W. Hanson and Liu Ge in the Department of Biochemistry at Case Western Reserve University School of Medicine. "These results provide an important mechanism to understand the efficient gene transfer capabilities of compacted DNA," said Dr. Hanson. "DNA nanoparticles may provide an effective platform to transfer therapeutic genes for a variety of human clinical diseases."

"Copernicus has established platform gene transfer and expression technologies that are effective and safe," said Robert C. Moen, M.D., Ph.D., President and CEO of Copernicus. "Our in vivo gene transfer efficiency in the airways of mice is comparable to those observed using viral vectors. This paper is an example of our desire to better understand the mechanisms responsible for our highly efficient gene transfer results. In contrast to viral vectors, our DNA nanoparticles are non-toxic, non-inflammatory, and non-immunogenic, and we believe that recurrent administration of our composition is possible. Our recent human clinical trial results in cystic fibrosis subjects were quite encouraging, and we are developing DNA nanoparticle aerosols for CF and other clinical indications."

Copernicus Therapeutics, Inc. is advancing novel targeting and delivery systems with broad applications in human therapeutics and vaccines. Copernicus' technologies include a multi-component delivery platform that can be applied to nucleic acids to develop therapies for a variety of human diseases and a targeting platform enabling the efficient uptake of drugs by specific cells and tissues. The Company's targeting and delivery platforms are complementary and can be combined to enhance the efficacy and safety of existing drugs or to create novel therapeutics.

Founded in 1843, the Case Western Reserve University School of Medicine is the largest medical research institution in Ohio and the 16th largest among the nation's medical schools for research funding from the National Institutes of Health. Seven Nobel Laureates have been affiliated with the school. The School of Medicine is recognized throughout the international medical community for outstanding achievements in research, teaching and service. Annually, the School of Medicine trains more than 600 M.D. and M.D./Ph.D. students.