Genprex, Inc., a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and other serious diseases, today announced that it signed an exclusive license agreement with the University of Pittsburgh for a diabetes gene therapy that may have the potential to cure Type 1 and Type 2 diabetes, which together currently affect approximately 30.3 million people in the U.S, or 9 percent of the U.S. population.
Developed by lead researcher, Harvard graduate, and McGowan Institute for Regenerative Medicine affiliated faculty member George Gittes, MD, the Director of the Richard King Mellon Foundation Institute for Pediatric Research and Co-Scientific Director at UPMC Children’s Hospital of Pittsburgh, the diabetes gene therapy works by reprogramming beta cells in the pancreas to restore their function, thereby replenishing levels of insulin. 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.
The diabetes gene therapy has been tested in vivo in mice and nonhuman primates. In studies of diabetic mice, the gene therapy approach restored normal blood glucose levels for an extended period of time, typically around four months. According to Dr. Gittes, the duration of restored blood glucose levels in mice could translate to decades in humans. Following preclinical studies, Dr. Gittes and his team plan to begin a Phase I clinical trial in diabetic patients, which could be the first-ever gene therapy tested in humans for diabetes.
“One of the biggest advantages of this gene therapy is that it could eliminate the need for insulin replacement therapy for diabetic patients,” said Dr. Gittes. “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. This therapy has the potential to truly disrupt the diabetes market.”
“We are excited to announce the licensing agreement with The University of Pittsburgh, and we look forward to working with Dr. Gittes and his team to develop this groundbreaking treatment for diabetes,” said Rodney Varner, Genprex’s Chairman and Chief Executive Officer. “At Genprex, we have always put patient needs first, focusing on ways to bring new treatment options to patient populations who have large unmet medical needs. We believe this diabetes gene therapy may potentially become a new treatment option for the millions of diabetes patients who now must take insulin replacement therapy, and it may be effective for patients who do not benefit sufficiently from that therapy. Even more moving, the diabetes gene therapy could hold the potential to provide long term effectiveness, or even be a cure, for diabetes patients.”
Genprex plans to pursue potential partnerships for the development of this therapy globally and in the U.S.
According to the American Diabetes Association, more than 30 million Americans have diabetes, and approximately 1.5 million Americans are diagnosed with diabetes every year. Diabetes patients have the continuous burden of checking and monitoring their blood glucose levels and injecting insulin on a daily basis. Without effective management of diabetes, patients are at risk of stroke, hyperglycemia, cardiovascular disease, diabetic ketoacidosis and extremity amputation. Diabetes is the seventh leading cause of death in the U.S.