GPX-002 and GPX-003

Diabetes Gene Therapies

GPX-002 and GPX-003, our diabetes gene therapy candidates, are the most recent additions of our licensed technologies. Both technologies were developed by researchers at the University of Pittsburgh.

GPX-002 for Type 1 Diabetes

GPX-002 is comprised of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector with a glucagon promoter 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.

GPX-002 has been tested in vivo in mice and nonhuman primates. Earlier studies in diabetic mouse models showed that an earlier version of GPX-002 restored normal blood glucose levels for an extended period of time, typically around four months. It is believed that the duration of restored blood glucose levels in mice 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.

Genprex recently announced a license agreement with the University of Pittsburgh for a preclinical technology that transforms macrophages enabling them to reduce autoimmune activity in Type 1 diabetes, a technology that could be complementary to the GPX-002 diabetes technology.

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.

GPX-003 for Type 2 Diabetes

GPX-003 is believed to work by rejuvenating diminished beta cells to increase insulin expression by introducing transcription factors controlled by an insulin promoter.

GPX-003 is based on the same general gene therapy approach under Genprex’s original license that is comprised of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes directly to the pancreas.

Both diabetes gene therapies were developed by Dr. George Gittes, Professor of Surgery and Pediatrics and Chief of the Division of Pediatric Surgery at the University of Pittsburgh School of Medicine, where preclinical research is ongoing. 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, GPX-003 and the Pdx1/MafA genes, please refer to our Clinical Trials and Pdx1/MafA Bibliography pages.