Genprex Announces Milestone Preclinical Proof-of-Concept for GPX-002 Diabetes AAV Gene Therapy in Non-Human Primates

AUSTIN, TX —January 6, 2026— Genprex, Inc. (NASDAQ: GNPX) today revealed breakthrough preliminary in vivo data for GPX-002, a potential “one-time” gene therapy for both Type 1 and Type 2 diabetes. The study successfully demonstrated that direct pancreatic duct infusion of the therapy normalized glucose levels in non-human primates (NHP) with severe Type 2 diabetes, marking a critical leap toward human clinical trials.

A Novel Approach: Rejuvenating the Pancreas

Unlike current treatments that focus on supplemental insulin, GPX-002 aims to restore the body’s natural insulin-producing capacity. The therapy uses an adeno-associated virus (AAV) vector to deliver two master transcription factor genes—Pdx1 and MafA—directly to the pancreas.

In the Type 2 diabetes (T2D) model, where beta cells typically become “exhausted” and fail to secrete insulin, GPX-002 acts to:

• Rejuvenate Beta Cells: Restores the function of failing beta cells, allowing them to properly release insulin in response to glucose.

• Normalize Glucose Tolerance: In the study, a severely diabetic NHP achieved normal glucose tolerance test results seven months post-infusion.

• Enable Endoscopic Delivery: In humans, this AAV delivery is designed to be performed via a routine, minimally invasive endoscopy.

Comparative Delivery Methods and Immune Management

The study compared two delivery methods in NHPs:

• Intraductal Infusion: Resulted in complete normalization of glucose tolerance, proving to be the highly favorable method.

• Direct Pancreas Injection: Showed significant efficacy but did not reach full normalization of glucose levels.

A key finding involved the management of the AAV immune response. Researchers discovered that a defined six-month course of immunosuppression prevented the body from rejecting the viral vector, after which the therapy remained stable and effective without further immune intervention.

Validation in Mouse Models

The NHP data follows successful results in T2D mice, where GPX-002 was shown to reverse hyperglycemia within four weeks. The mice demonstrated a statistically significant increase in glucose-stimulated insulin secretion, confirming the therapy’s ability to replenish the beta cell population.

“This work demonstrates the potential to unlock treatment opportunities for the 90 to 95 percent of patients afflicted by Type 2 diabetes,” said Ryan Confer, CEO of Genprex. The company is now moving toward formal toxicology studies and a subsequent Investigational New Drug (IND) submission to the FDA.