Copernicus Therapeutics, Inc. has announced that a research team at University of Oklahoma Health Sciences Center, led by Dr. Muna Naash, professor of Cell Biology, has demonstrated that Copernicus’ DNA nanoparticles safely deliver and express DNA in the rods and cones of the mouse retina.
According to Dr. Naash’s team, current data indicate that greater than 95% of these retinal cells expressed the DNA nanoparticle and there was no evidence of toxicity.
These findings, published on December 20, 2006 in the journal PLoS ONE, have implications for the development of DNA-based therapeutics for various eye disorders, including retinitis pigmentosa and macular degeneration company officials said.
“These exciting results suggest that genetic replacement therapy is feasible for various eye diseases,” said Robert C. Moen, M.D., Ph.D., president and CEO of Copernicus.
“The Copernicus DNA nanoparticle formulation is safe and effective and permits a non-viral approach to treat human disease by introducing a normal copy of the underlying gene that is responsible for the disease process,” Dr. Moen continued.
“In addition to corrective therapy for genetic diseases such as retinitis pigmentosa, nucleic acid nanoparticles may provide effective treatments for more complex disorders such as diabetic retinopathy, macular degeneration, and various diseases that injure ganglion cells and the optic nerve.”
“We are pleased by the promise of the results and look forward to continuing our work in this area,” said Dr. Naash. “Dr. Muna Naash’s work underscores the importance of corporate and university-based investigators working together to explore new and potentially effective treatments for human blinding disorders,” Dr. Moen said.