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AUSTIN, Texas & CAMBRIDGE, Mass.—Type 1 and type 2 diabetes affect about 9 percent of the U.S. population alone—equivalent to roughly 30 million people. To help bring that number down, Genprex Inc.—a clinical-stage gene therapy company that develops technologies for patients with cancer and other serious diseases—recently signed an exclusive license agreement with the University of Pittsburgh for a diabetes gene therapy aimed at curing type 1 and type 2 diabetes.
“The diabetes gene therapy, which was developed by lead researcher and Harvard graduate Dr. George Gittes at the Rangos Research Center at UPMC Children’s Hospital of Pittsburgh, works by reprogramming beta cells in the pancreas to restore their function, thereby replenishing levels of insulin,” explained Rodney Varner, Genprex’s chairman and CEO. “The novel infusion process uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. The proteins these genes express transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body’s immune system.”
He added, “In a diabetic mouse model, the newly reprogrammed beta-like cells restored normal blood glucose levels for an extended period of time. The functional beta-like cells are able to produce insulin, but are distinct enough from beta cells to evade the immune system. This therapy and process could eliminate the need for insulin replacement therapy in humans.”
As Gittes explained, “Lifting this huge burden for the millions of patients who must continuously monitor blood glucose levels and inject insulin daily would be a breakthrough in modern medicine.”
Varner believes that this diabetes gene therapy could become a new treatment modality for the millions of diabetes patients who now must take insulin replacement therapy, and it could also be effective for patients who do not benefit sufficiently from that therapy. He added, “The diabetes gene therapy could hold the potential to provide long-term effectiveness, or even be a cure, for diabetes patients.”
The diabetes gene therapy candidate has undergone testing in-vivo in mice and nonhuman primates. In studies of diabetic mice, the gene therapy approach restored normal blood glucose levels for about four months. According to Gittes, the duration of restored blood glucose levels in mice could mean decades in humans.
Genprex plans to add this technology to its research and development pipeline, diversifying its portfolio and expanding its clinical development programs. The company will continue its focus on developing its immunogene therapies for cancer, including Oncoprex immunogene therapy, its lead drug candidate for non-small cell lung cancer, in parallel with development of the new diabetes gene therapy.
Varner said that the diabetes gene therapy candidate is still in a preclinical phase, with mouse and primate studies that are currently ongoing. While Gittes and his team are planning to begin a Phase 1 trial in diabetic patients, more safety studies are needed before this approach moves into the clinic.
Varner added, “Genprex plans to continue studying the technology in a preclinical phase at the University of Pittsburgh. As this group continues to refine the research, then we will move forward with a clinical trial when sufficient animal data has been generated. The Phase 1 clinical trial in diabetic patients could be the first-ever gene therapy tested in humans for diabetes.”
As for commercial potential for this gene therapy, the global diabetes drugs market was valued at $48 billion in 2018, and it is projected to reach $78 billion by the end of 2026, exhibiting a compound annual growth rate of 6.1 percent, according to industry reports.
“Genprex is also pursuing potential partnerships for the development of this therapy globally and in the U.S.,” Varner said. “This therapy has the potential to truly disrupt the diabetes market.”