Gene Therapy Milestone: Genprex's GPX-002 Demonstrates Insulin Production Recovery in Type 2 Diabetes Models

Genprex Inc. (GNPX) has announced breakthrough findings for its investigational gene therapy GPX-002, marking a significant stride in addressing Type 2 diabetes through cellular rejuvenation. The company’s preclinical studies in both non-human primates and mice have provided compelling evidence that the therapy can restore insulin-producing capacity in damaged beta cells—a hallmark of Type 2 diabetes pathology.

The Technology Behind GPX-002

At its core, GPX-002 employs an innovative delivery approach: an adeno-associated virus (AAV) vector carries the Pdx1 and MafA genes directly into the pancreatic duct. This targeted gene delivery mechanism is designed to address the fundamental problem in Type 2 diabetes: beta cells progressively lose their ability to produce and secrete insulin. By introducing these specific genes, the therapy aims to reprogram and reactivate these exhausted cells, essentially reversing the disease’s cellular damage.

Primate Studies Show Remarkable Response

The non-human primate research yielded particularly encouraging results. The first subject received GPX-002 through an intraductal infusion method—a technique that allows the therapeutic genes to reach target cells within the pancreas. Within seven months, this animal progressed from severe baseline Type 2 diabetes to demonstrating normal glucose tolerance, a dramatic reversal of what is typically an irreversible metabolic disorder.

A second primate underwent treatment via direct pancreatic injection and showed substantial improvement, though complete normalization was not achieved. These differential outcomes suggest that the intraductal infusion delivery method may offer superior efficacy for targeting the affected cell populations.

Researchers observed that subjects receiving AAV-based therapy required temporary immunosuppression during treatment. Importantly, immune responses were noted to decline after approximately six months, potentially opening doors for refined protocols that could improve long-term tolerability and outcomes.

Rodent Model Confirms Mechanism

Supporting the primate findings, GPX-002 demonstrated robust activity in Type 2 diabetic mice. Treated animals exhibited elevated glucose-stimulated insulin secretion and complete reversal of hyperglycemia—with blood glucose levels normalizing within four weeks of treatment. These accelerated timelines in rodents further validate that the therapy successfully reverses beta cell dysfunction across multiple disease models.

Expanding Beyond Type 1 Diabetes

Earlier GPX-002 studies in Type 1 diabetes models showed the therapy could convert alpha cells into functional beta-like cells capable of producing insulin. These new Type 2 results represent a critical expansion of the candidate’s scope, given that Type 2 diabetes affects a vastly larger patient population than Type 1. This multiplied addressable market significantly enhances the potential commercial impact.

Clinical Path Forward

Genprex is advancing multiple next-stage studies, continuing expanded non-human primate research in both diabetes types while simultaneously preparing formal toxicology assessments. These regulatory milestones are designed to support a future Investigational New Drug application, setting the stage for human trials.

Market Response

The market has taken notice of these developments. GNPX shares closed the previous trading session at $1.80, representing a 0.86% gain, and entered pre-market trading at $1.90—a 5.55% increase that reflects investor optimism around the therapy’s potential to address one of the most prevalent metabolic disorders globally.