Microfluidic - Gene Therapy

Gene therapy is an incredibly powerful technology that is currently used to treat and even cure rare diseases and select types of cancer. One such treatment involves removing cells from a patient; altering the patient’s genes in the isolated cells through viral vectors; and administering the manipulated cells back to the patient. Gene and cell-based therapies require highly efficient methods that do not harm a patient's cells during the process. According to the National Heart, Lung, and Blood Institute, gene therapy could be used to treat more common conditions like cancer or infections in the future. Unfortunately, these technologies are inaccessible to many, as the viral delivery aspect can cost millions of dollars.

While viral delivery has been approved by the FDA, several cases have shown patients develop secondary conditions from this method. Viral delivery is inconsistent and can be harmful to its recipients.

Guigen Zhang, Ph.D, and colleague, Sheng Tong, Ph.D., have developed a technology that avoids the viral delivery aspect of gene therapy. Their innovation uses a less harmful procedure, ensuring better cell health while delivering the highest possible efficiency with consistency at the individual cell level, allowing a treatment procedure to have a higher likelihood of success. This technology involves a low-cost 3D microfluidic approach to deliver therapeutic genes into cells in a continuous high-throughput manner.

Zhang’s main goal is to provide an affordable and accessible solution to gene therapy. Zhang stresses, in order for gene therapy to be economical, it should be used for other diseases that may be difficult to treat, not just rare conditions. His goal is to help improve people’s quality of life and also allow them to lead productive lives even in old age. He hopes that their technology will help to make gene therapy an affordable and accessible medical procedure.

Zhang is a biomedical engineering professor at the University of Kentucky. He is a fellow of American Institute of Medical and Biological Engineering, fellow of Biomaterials Science and Engineering, and fellow of Biomedical Engineering Society. After getting the Biomedical Engineering undergraduate program off the ground at UK while serving as the Chair of Biomedical Engineering at UK, he now devotes his research to advancing medical technologies to help people.

This technology is still in the early stages, but it proves to be promising. Zhang and his team have conducted preliminary tests which have had almost 100 percent transfection efficiency. They are currently in the process of seeking funding, including NIH grants, to further develop the technology.

When Zhang started working with Launch Blue and the University of Kentucky Office of Technology Commercialization (UK OTC), he was focused on an entirely different aspect of the gene therapy process. He originally hoped to apply the technology to a preceding step of the therapy, isolating the cells, but realized through his customer discovery efforts that it would be better utilized for delivering genes into cells without using viral vectors.

With support from UK OTC and Launch Blue, Zhang traveled to New Orleans to present this technology at “BIO on the BAYOU”, a conference for innovators in the science and biotech industry to showcase their technologies. At the conference, Zhang was able to connect with others within the same field, who showed a lot of interest in his technology. He was also able to connect with other researchers who are in urgent need of a non-viral high-throughput gene delivery technology. He is currently in the process of establishing a collaboration with a colleague at Tulane National Primate Lab, one of the seven primate labs in the country, on testing this microfluidic cell transfection device in treating HIV using rhesus monkeys.

It might be easier to attract funding for this type of technology in the Bay Area on the West Coast, or in Boston on the East Coast, but Dr. Zhang hopes that the KYNETIC program can help advance this technology in Kentucky, so it can help people sooner.

“I don’t want to give people the impression that this technology is ready, we’re not there yet,” Zhang explained, “We have all this anticipated capability that could bring change throughout the fields, but we need support to help us get there.”

Zhang believes that this promising technology will someday make gene therapy affordable and accessible to all, especially those in urgent need, but without any means to pay for it. He also believes that a cost effective non-viral delivery method can help make this a reality sooner.

By: Lauren Balla

Launch Blue nurtures promising startup founders and university innovators through intensive accelerator and incubator programs. Its funding partners are the University of Kentucky: Office of Technology Commercialization, KY Innovation, the U.S. Economic Development Administration, and the National Science Foundation.