On April 28, 2026 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that its research collaborators will present positive preclinical data on the Company’s diabetes gene therapy drug candidate at the upcoming 2026 American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, taking place May 11-15, 2026 in Boston, Mass. The collaborators will present preclinical data demonstrating that the diabetes gene therapy (Pdx1/MafA gene therapy, PM or GPX-002) can reverse hyperglycemia in Type 2 diabetic (T2D) mouse models.
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"Our collaborators’ preclinical data to be presented at ASGCT (Free ASGCT Whitepaper) 2026 demonstrate the ability of our diabetes gene therapy to reverse hyperglycemia in T2D models, representing a significant advancement toward a novel therapeutic paradigm for T2D patients," said Ryan Confer, President and Chief Executive Officer at Genprex. "The compelling evidence from the preclinical studies, which achieved complete rescue of HFD-induced hyperglycemia at four weeks post-treatment via direct intrapancreatic infusion, suggests the technical translatability of our diabetes gene therapy approach to human application, potentially through endoscopic retrograde cholangiopancreatography, offering a potentially promising avenue for long-term glycemic control in T2D."
The featured Genprex-supported abstract and poster at the 2026 ASGCT (Free ASGCT Whitepaper) Annual Meeting:
Title: "Pancreatic Delivery of AAV-Pdx1/MafA Reverses Hyperglycemia in a Preclinical Model of Type 2 Diabetes"
Abstract ID: 2419
Topic: Gene-Based Therapies in Pre-Clinical Models of Genetic Disease
Poster Presentation Date: Wednesday, May 13, 2026
Poster Presentation Time: 5-6:30 p.m. ET
In this study, eight-week-old male C57BL/6 mice were maintained on a regular diet (RD) or high fat diet (HFD) for 24 weeks. HFD mice then either remained unoperated or underwent intrapancreatic infusion of adeno-associated virus (AAV-8) encoding Pdx1 and MafA (PM) cassettes under the CMV promoter (global–islet cell targeting) or the rat insulin promoter (RIP) (β-cell–specific targeting) or received a control virus. The diet remained unchanged after surgery. At two and/or four weeks after surgery, researchers performed intraperitoneal glucose tolerance testing (IPGTT), insulin tolerance testing (ITT), glucose-stimulated insulin secretion (GSIS), calculated HOMA-IR and assessed glucagon secretion. Mice were then euthanized for pancreatic histology, quantification of β- and α-cell mass, electron microscopy (EM), and islets were isolated for ex-vivo glucose-stimulated insulin secretion (GSIS) and single-cell RNA sequencing. The results at four weeks showed major improvements in the control of diabetes.
At four weeks after surgery, ex-vivo GSIS showed that islets isolated from HFD+CMV-PM-GFP treated mice had insulin secretion similar to islets from RD mice, and both groups had increased insulin secretion compared to islets from the control HFD groups, indicating improved β-cell function with PM treatment.
Similarly, and importantly, treatment of HFD mice with RIP-PM-GFP, which selectively targets β-cells, reversed hyperglycemia and improved ex-vivo GSIS. In addition, EM imaging showed that PM treatment in HFD mice increased the number of total and mature insulin granules and decreased the number of immature insulin granules compared with HFD controls. Furthermore, transcriptomic pseudotime analysis demonstrated a shift in β-cells from an immature state toward a more mature state after PM treatment.
PM gene therapy reverses hyperglycemia, likely in large part by specifically enhancing β-cell function and maturation. This approach is technically translatable to humans using endoscopic retrograde cholangiopancreatography to deliver PM gene therapy to the pancreas.
(Press release, Genprex, APR 28, 2026, View Source [SID1234664855])