Gene Therapy Approach Shows Promise in Restoring Insulin Production for Type 2 Diabetes

Genprex Inc. (GNPX) has unveiled encouraging preclinical findings for its investigational gene therapy candidate GPX-002, demonstrating functional restoration of insulin-secreting capacity in disease models. The breakthrough centers on a novel approach to address the fundamental problem in Type 2 diabetes: the progressive loss of beta cell function and insulin production capability.

The Science Behind GPX-002

The therapeutic mechanism involves delivering two key genetic factors—Pdx1 and MafA genes—directly into the pancreatic duct using an adeno-associated virus (AAV) vector. This delivery strategy targets the root cause of Type 2 diabetes by attempting to reprogram dysfunctional beta cells, restoring their natural ability to manufacture and release insulin in response to blood glucose fluctuations.

The gene therapy approach builds on the principle that beta cells in Type 2 diabetes don’t necessarily die—they become exhausted and lose their productive capacity. GPX-002 is designed to reverse this exhaustion rather than replace the cells entirely.

Non-Human Primate Study Results

The most compelling evidence emerged from primate studies. The first animal received GPX-002 via intraductal infusion and experienced progressive glucose tolerance improvements throughout a seven-month observation period, ultimately achieving normal glucose regulation despite having severe Type 2 diabetes at baseline. A second primate treated through direct pancreatic injection also demonstrated substantial improvement, though fell short of complete normalization.

Genprex researchers identified intraductal infusion as the potentially superior delivery method for reaching both alpha and beta cell populations. However, they noted that AAV-based therapies in primates require temporary immunosuppression, with immune responses typically normalizing after approximately six months. This timeline suggests future protocols could optimize immunosuppression strategies to enhance therapeutic outcomes.

Mouse Model Confirmation

Parallel studies in Type 2 diabetic mice reinforced the primate findings. Treated animals exhibited enhanced glucose-stimulated insulin secretion and reversal of elevated blood glucose levels, with glucose normalization occurring within four weeks post-treatment. These rapid results further validate beta cell rejuvenation as the underlying mechanism.

Broader Development Context

The current findings extend earlier research in Type 1 diabetes models, where GPX-002 demonstrated the capacity to convert dysfunctional alpha cells into functional beta-like cells capable of insulin synthesis. The expansion to Type 2 diabetes represents a significant opportunity, given the substantially larger patient population affected by the Type 2 form of the disease.

Genprex is advancing multiple work streams, continuing non-human primate studies across both diabetes types and initiating formal toxicology assessments in preparation for a future Investigational New Drug application with regulatory authorities.

Market Response

GNPX shares closed at $1.80, representing a 0.86% gain, and subsequently moved to $1.90 in pre-market trading, reflecting a 5.55% increase. The stock movement reflects investor interest in the early-stage therapeutic progress.