Most diseases and disorders are the results of harmful proteins that are produced by genes that are faulty. Infectious agents, such as viruses, invade cells and take control of the DNA apparatus as a way of reproducing and killing or damaging cells. Genetic predisposition or environmental factors can also trigger faulty DNA that can cause many disorders such as Cancer and Alzheimer's disease.
Most pharmacological agents act by chemically signaling cells to interrupt or reduce production of the “bad” protein, or by binding to and adjusting the structure of a protein to ameliorate its activity. Traditionally, these agents were discovered by accident or serendipity. Most drugs are systemic and usually cause side effects.Aside from vaccines, few of today's drugs are curative or preventative.
It is estimated that there are approximately 35,000 human genes, each coding for a different protein. These genes may suffer mutations leading to production of faulty or harmful proteins. Although the molecular nature of many diseases is not yet understood, thousands of researchers in academia, government and industry are working to identify and characterize disease related proteins and genes. Their work adds to the growing body of knowledge about specific disease mechanisms. Once these disease gene targets are identified, validated, and understood as to their function, then therapeutic agents can be designed to halt, reduce, or correct their disease causing action.
This constitutes a technological revolution and an economic imperative for large pharmaceutical companies who need a steady flow of new drugs in their product pipelines while reducing the immense cost of drug discovery failure.
For thirty years, scientists have postulated that an effective disease fighting strategy is to change genes directly in cells of living beings. Over the past ten years, the field of gene therapy has developed. Today the viability of this treatment is still in question principally because of the lack of robust delivery mechanisms. Never the less, gene therapy is in the mainstream of experimental medicine and investigators in major medical academic centers are conducting clinical trials on patients with difficult to treat diseases.In industry, several biotechnology companies are developing gene-altering compounds.
In its basic form, the protein generating mechanism of cells is a biochemical process that starts with the transcription of DNA instructions into messenger RNA (mRNA) followed by the translation of the mRNA into the building blocks of proteins.
CytoGenix proprietary technology¹ interrupts the translation of mRNA.This occurs through a novel method of introducing specific DNA instructions that direct the cell's machinery to cancel translation of the mRNA of a specified gene into a protein.By harnessing the cell's natural internal DNA machinery to replicate the gene blocking instructions, this technology overcomes the limitation of other gene medicine approaches that require the introduction of large concentrations of DNA into the cells from external sources.
This technology enables and enhances the performance of gene medicine methods such as antisense and triplex.This technology also has great promise as a platform for the recently discovered DNA enzyme method.This fundamental molecular control in cells has the potential to be widely applied for the development of therapeutic agents against certain cancers, autoimmune and infectious diseases.The Company is developing various therapeutic products for diseases of the skin.The Company intends to seek licensees for other disease targets.
¹U.S. patent # 6,054,299
