Diabetes Gene Therapy

GPX-002, a gene therapy for diabetes, is the most recent addition of our licensed technologies. GPX-002 was developed by researchers at the University of Pittsburgh. Diabetic mice studies have shown that GPX-002 restored normal blood glucose levels for an extended period of time, which could translate to decades in humans. This gene therapy could not only become a new treatment option for millions of diabetes patients who need insulin replacement therapy, but it holds the potential to provide long-term effectiveness, or may even be a cure, for diabetic patients.

The diabetes gene therapy, GPX-002, is comprised of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. The genes express proteins that 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.

Image source: Osipovich, Anna & Magnuson, Mark. (2018). Alpha to Beta Cell Reprogramming: Stepping toward a New Treatment for Diabetes. Cell Stem Cell. 22. 12-13. 10.1016/j.stem.2017.12.012.

Diabetic mice studies show that the gene therapy restored normal blood glucose levels for an extended period of time, typically around four months. The duration of restored blood glucose levels in mice could translate to decades in humans.

The diabetes gene therapy was developed by Dr. George Gittes, a researcher at the Rangos Research Center at UPMC Children’s Hospital of Pittsburgh, where preclinical research is ongoing. GPX-002 has been tested in vivo in mice and nonhuman primates. Once sufficient preclinical data has been generated, we expect to begin a Phase I clinical trial in diabetic patients, which could be the first-ever gene therapy tested in humans for diabetes.

To learn more about scientific evidence and studies supporting GPX-002 and the Pdx1/MafA genes, please refer to our Clinical Trials and Pdx1/MafA Bibliography pages.