Clinical Trials


Studying GPX-002 and GPX-002 in Diabetic Mice and Non-Human Primates

Researchers at the University of Pittsburgh have conducted preclinical studies evaluating GPX-002 in diabetic mice and non-human primates. In vivo  studies have found that GPX-002 restored normal blood glucose levels for an extended period of time, which lasted approximately four months. According to the researchers, the duration of restored blood glucose levels in mice could translate to decades in humans.

In August 2022, we entered into a one-year sponsored research agreement with the University of Pittsburgh for the use of GPX-003 in a non-human primate model in Type 2 diabetes.

In February 2023, research collaborators at the University of Pittsburgh presented preclinical data in a NHP model of Type 1 diabetes highlighting the therapeutic potential of GPX-002 The statistically significant study results showed that after infusion of the AAV engineered construct all eight of the NHPs had:

  • Decreased insulin requirements (p<0.001);
  • Increased c-peptide levels (p<0.05);
  • Glucose tolerance compared to baseline (p<0.05) with one demonstrating reestablished normoglycemia; and
  • Insulin and glucagon staining in the same cells, suggesting the formation of insulin-producing cells.

We believe the data in NHPs demonstrate the potential for this gene therapy treatment to eliminate the need for insulin replacement therapy for Type 1 and Type 2 diabetic patients.

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