GPX-002 and GPX-003

Diabetes Gene Therapies

In diabetes, we have exclusively licensed from the University of Pittsburgh multiple technologies relating to the development of a gene therapy product for each of Type 1 and Type 2 diabetes.

The same general novel approach is used in each of Type 1 and Type 2 whereby an adeno-associated virus (AAV) vector containing the Pdx1 and MafA genes is administered directly into the pancreatic duct. In humans, this can be done with a routine endoscopy procedure. Our diabetes product candidates are currently being evaluated and optimized in preclinical studies at the University of Pittsburgh.

GPX-002 and GPX-003

GPX-002 is designed to work in Type 1 diabetes by transforming alpha cells in the pancreas into functional beta-like cells, which can produce insulin but may be 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.

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, which lasted approximately four months, and markedly increased the mass of insulin producing beta cells. According to the researchers, the duration of restored blood glucose levels in mice could potentially translate to decades in humans. 

In Type 2 diabetes, GPX-003 is believed to work by replenishing and rejuvenating the beta cells that make insulin.

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 (NHP) model in Type 2 diabetes.

In February 2023, the Company’s 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 at the 16th International Conference on Advanced Technologies & Treatments for Diabetes (ATTD 2023) in Berlin, Germany. 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);
  • Improved 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, GPX-003 and the Pdx1/MafA genes, please refer to our Clinical Trials and Pdx1/MafA Bibliography pages.