Copernicus’ Nanoparticle Technology Applied to siRNA
News Jun 07, 2007
Copernicus Therapeutics, Inc. has announced that at the American Society of Gene Therapy (ASGT) Meeting held in Seattle, Washington, Copernicus showed that the methods used to produce DNA nanoparticles were also very effective when applied to siRNA.
Both DNA nanoparticles as well as nanoparticles made with siRNA are highly resistant to degradative processes that would destroy the therapeutic benefit of the nanoparticles. These findings are significant because siRNAs can be designed to block the expression of specific genes and have the potential to treat numerous human diseases, including viral infections. A 2006 Nobel Prize was awarded to the scientists that discovered siRNA.
“We are most enthusiastic about these findings,” said Dr. Mark J. Cooper, Sr. Vice President of Science and Medical Affairs at Copernicus.
“Our non-viral nucleic acid delivery system provides important safety and efficacy characteristics not seen with viral-based systems. We have extensive experience in the development of DNA nanoparticles, including an encouraging clinical trial in subjects with cystic fibrosis. The data presented at ASGT show that we may be able to expand the use of our non-viral nucleic acid delivery system to the exciting field of siRNA. These results have encouraged us and others to explore the use of our siRNA delivery platform to treat a variety of serious diseases,” said Cooper.
“These findings further demonstrate the platform nature of our nucleic acid nanoparticle technology which enables us to develop nucleic acid therapeutics for different parts of the body and for multiple diseases,” said Dr. Robert C. Moen, President and CEO of Copernicus.
“Our lead DNA nanoparticle program is developing a therapy for a serious disease affecting the lung, cystic fibrosis. Opportunities for siRNA nanoparticles are numerous, and include serious viral lung infections and other genetic disorders,” Dr. Moen added.
As genome editing technologies advance toward clinical therapies, they are raising hopes of a completely new way to treat disease. However, challenges need to be addressed before potential treatments can be widely used in patients. To tackle these challenges, the National Institutes of Health has launched the Somatic Cell Genome Editing program, which has awarded multiple grants including more than $3.6 million to assess the safety of genome editing in human cells and tissues.