As cells constantly process genes, there may be instances where a gene becomes defective and detrimental as it is unable to create proteins essential for normal body function. The goal of our gene therapy platform is to correct these defective genes by delivering a functional copy to target cells, where they may incorporate this copy and repair their function.
We utilize viruses such as adenoviruses and lentiviruses, which have been modified so that they do not cause disease. The well-studied mechanisms used by viruses to infiltrate cells make them a common vector in gene therapy. It is our belief that we may be able to incorporate methods infamous from a public health perspective into a strategy in repairing defective genes.
Our gene therapy vectors have been shown to deliver genes with a high degree of efficiency and specificity, maximizing the chances in which our therapy can successfully resolve gene defects. By utilizing our platform, we offer excellence through three principles: transduction efficiency, expression efficiency, and controllability.
We have achieved transduction efficiency in the development of our vectors. A lower initial concentration of vectors is required to start the gene therapy process. Like the viral structures they emulate, our gene therapy technology utilizes the cell’s natural mechanisms to produce more copies. This establishes a self-sustaining vector concentration that can proliferate within intracellular conditions and continue its function until the target gene has been repaired.
We offer expression efficiency which continues to be refined over time through our ongoing research. Some vectors may be more efficient at modifying gene expression under certain conditions. Through our research, we have identified optimized vectors for use in any individual, using clinical data and neural network predictions made from analyzing the subject’s genome. This allows us to provide highly customized gene therapy vectors, tailored specifically to the individual.
Controllability is an important feature in our vector design. This includes the establishment of conditions in which the vector starts and stops regulating epigenetic factors. Our adenovirus vectors do not naturally replicate within the human body, thus allowing for a controlled amount of gene therapy to be administered. Ultimately, the optimized controllability of our platform allows for it to be tapered down accordingly as needed and prevent the emergence of adverse reactions and undesired side effects.