GPX-002
GPX-002 For Type 1 and Type 2 Diabetes
In our novel gene therapy diabetes program, we have exclusively licensed from the University of Pittsburgh multiple technologies relating to the development of a gene therapy product for both Type 1 and Type 2 diabetes.
GPX-002 is being developed using the same construct for the treatment of both Type 1 diabetes and Type 2 diabetes (formerly known as GPX-003 when a different construct was being considered for Type 2 diabetes). The same general novel approach is used in each of Type 1 and Type 2 diabetes 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.
In Type 1 diabetes, GPX-002 is designed to work 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. In vivo, preclinical studies show that GPX-002 restored normal blood glucose levels for an extended period of time.
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 at the 16th International Conference on Advanced Technologies & Treatments for Diabetes (ATTD 2023) in Berlin, Germany.
The statistically significant study results showed the treated animals had:
- Decreased insulin requirements
- Increased c-peptide levels
- Improved glucose tolerance compared to baseline
In April 2023, Genprex hosted a Key Opinion Leader virtual event entitled “Novel Gene Therapy to Treat Type 1 Diabetes,” which discussed preclinical data reported at ATTD 2023 supporting gene therapy to treat Type 1 diabetes. Watch the KOL event here.
In a similar approach for Type 2 diabetes, GPX-002, where autoimmunity is not at play, is believed to work by replenishing and rejuvenating exhausted beta cells that make insulin.
To learn more about scientific evidence and studies supporting GPX-002 and the Pdx1/MafA genes, please refer to our Pdx1/MafA Bibliography page.
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