|Copernicus Therapeutics, Inc.
Delivering the promise of nucleic acid therapeutics
The last few decades have seen scientists and clinicians rapidly develop an understanding of the basis of many human diseases at the molecular level. Concerted efforts, such as the multi-billion dollar Human Genome Project, have helped provide this information leap. With a better understanding of the molecular basis of many diseases, it has been possible to envision new approaches and drugs to treat previously poorly treated or untreatable diseases. One of the new approaches has been to use the information obtained in the Human Genome itself to develop treatments. Nucleic acids are molecules our bodies use to store and transmit all the information that permits our bodies, organs, tissues and cells to function normally. With the new knowledge obtained and tools developed by scientists, it is now possible for clinician-scientists to devise ways to use nucleic acids as a new class of drugs.
|There are many ways to use nucleic acids as drugs; some are listed below:
- It is possible to deliver to cells nucleic acid molecules that serve as a blueprint to make proteins that replace defective proteins. These proteins can be correct copies of defective genes like the CFTR gene that, when mutated, causes cystic fibrosis, the most common monogenetic defect found in Caucasians. This use is called gene replacement therapy.
- Another possibility is to deliver to cells nucleic acid molecules that serve as the blueprint to make proteins which provide therapeutic function. Instead of having to make the protein in a manufacturing facility, the physician would provide the DNA to the patient's cells so the patient's own cells can now produce the needed therapeutic protein. Delivering a gene to encourage blood vessel formation in a heart to treat ischemia or delivering a gene to cancer cells that causes cells to produce a protein with anti-cancer properties are examples of this kind of therapy called therapeutic gene therapy.
- Nucleic acids may be delivered to cells that provide the code to turn off genes expressed in the patient. A variety of strategies are developed including anti-sense molecules, ribozymes, and siRNA. The latter two discoveries resulted in Nobel prizes. These types of nucleic acid molecules can be used to control viral infections like influenza. They could also be used to turn off abnormal blood vessel formation that cancer cells need to feed their destructive growth or stop the abnormal blood vessel formation that forms in many causes of blindness. This kind of therapy is called gene knockdown therapy.
|It is widely accepted that nucleic acid therapeutics provide great promise in treating human and animal disease, including diseases that may only respond to nucleic acid therapeutics. Most efforts to date have utilized viruses that have been altered to deliver the nucleic acid drug; these efforts have shown promise but have also shown the limitations of viral based systems. These limitations have included:
Just as Nicolaus Copernicus gave the world a different way to look at the solar system, Copernicus has taken a different view at delivering nucleic acid drugs. Copernicus developed a non-viral delivery system which avoids the limitations of the viral-based approaches. Others have tried non-viral approaches but often these trials result in delivery systems with toxicity, inefficiency, and/or stability problems. Copernicus believes the company has overcome these difficulties and developed a non-viral delivery system suitable for the safe and non-toxic delivery of nucleic acid drugs that potentially permit new therapies for a large variety of human diseases.
- Lack of efficient delivery of nucleic acid drugs
- Toxicity related to the delivery system itself. In a widely publicized case one patient died from such toxicity.
- Toxicity related to the mechanism used by the virus to deliver the nucleic acid drug. Several children in Europe treated with a viral-based delivery system developed cancer because of the viral system.
- The body's natural tendency to develop immune responses to the viruses used resulting in increased toxicity or decreased efficacy upon repeat dosing.
- The difficulty and costs associated with manufacturing viral-based nucleic acid drugs.
On this site you can find more information about the technology behind our breakthrough delivery system, clinical indications we have initially selected for further development, business aspects of our company, news and literature about our company, and the people that make up the dedicated employees of Copernicus.