Nucleic Acid Nanoparticles for Treating Human Diseases
Copernicus Business Strategy Overview
Copernicus Therapeutics, Inc. is a clinical stage biopharmaceutical company with its main focus upon developing its unique nucleic acid delivery and expression technologies for treating the root cause of cystic fibrosis. Exploiting its proprietary and complementary technologies, Copernicus has developed a non-viral nucleic acid delivery platform that permits the rapid design of nucleic acid drugs. This custom design of nucleic acid delivery vectors has significant efficacy, safety, and time to market advantages over vectors used by others. As an example of the power of Copernicus’ technologies, its lead product for treating cystic fibrosis has shown encouraging results in a clinical trial that we believe demonstrate superior safety and efficacy parameters when compared to others.
The business strategy of Copernicus is to build value by focusing on its core competencies, thus positioning Copernicus to become the preferred partner of pharmaceutical and biotechnology companies holding rights to genomic information and therapeutic genes that would benefit from access to the nanoparticle delivery technology. The outcome of this strategy is to provide Copernicus with revenue streams obtained from research, development, and manufacturing agreements from corporate partners. Since all the first generation products use the same raw materials, plasmid DNA and pegylated lysine peptides, the manufacturing process is the same. Copernicus intends to leverage our core competency to provide additional sources of revenue. Copernicus can provide assistance from discovery to production. Each product developed utilizes the same modular Copernicus platform technologies. This modular nature reduces the risk of development and permits Copernicus to perform the needed tasks faster, smarter, and for less cost.
The ability to use non-viral vectors to transfer DNA drugs to non-dividing cells is unique to Copernicus. Copernicus has outstanding opportunities available to it and its future partners in the following fields:
• Cystic Fibrosis – Delivery of DNA drugs to the lung by aerosols. An initial clinical trial in subjects with CF was encouraging and Copernicus is seeking funding to move this lead program forward.
• Eye diseases – Delivery of nucleic acid therapeutics is enabled using Copernicus’ non-viral delivery technology. Proof of concept in animal models of eye disease have been published.
• Brain Diseases – Copernicus has published proof of concept studies demonstrating that delivery of a therapeutic gene in an animal model of Parkinson’s disease has benefit. Delivery can be after a needle injection into the appropriate part of the brain or as simple as nasal installation of DNA nanoparticles.
• Lung diseases – The CF program has demonstrated our ability for delivery to lung cells. Delivery of therapeutic nucleic acids to treat and/or prevent other serious lung diseases is possible with our technology.