Genprex Collaborators Present Positive Preclinical Data on Diabetes Gene Therapy for Type 2 Diabetes at the 2026 American Society of Gene and Cell Therapy Annual Meeting
Rhea-AI Summary
Genprex (NASDAQ:GNPX) reported positive preclinical data for its diabetes gene therapy candidate Pdx1/MafA (PM, GPX-002) in Type 2 diabetes mouse models, presented at the 2026 ASGCT Annual Meeting.
Pancreatic AAV-Pdx1/MafA delivery reversed hyperglycemia within four weeks and improved β-cell function, insulin secretion, and maturation markers in high-fat-diet mice.
AI-generated analysis. Not financial advice.
Positive
- Pdx1/MafA (GPX-002) reversed hyperglycemia in Type 2 diabetic mouse models within four weeks
- PM-treated mice showed ex-vivo insulin secretion similar to regular-diet controls, indicating enhanced β-cell function
- β-cell–targeted RIP-PM-GFP improved ex-vivo GSIS and reversed hyperglycemia in high-fat-diet mice
- Electron microscopy showed increased total and mature insulin granules and fewer immature granules after PM treatment
- Transcriptomic pseudotime analysis indicated β-cells shifted from immature toward more mature states with PM therapy
- Approach is described as technically translatable to humans using endoscopic retrograde cholangiopancreatography delivery
Negative
- Results are limited to preclinical mouse models; no human clinical data are reported
- Long-term durability, safety, and efficacy of Pdx1/MafA gene therapy remain uncharacterized in this dataset
Key Figures
Market Reality Check
Peers on Argus
GNPX was up 2.54% pre-news on very light volume while peers showed mixed moves: CELZ -5.15%, CLDI +5.29%, INAB -1.85%, KPRX +11.16%, RNAZ +1.6%. This pattern points to stock-specific dynamics rather than a unified sector move.
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Apr 30 | Patent grant | Positive | +12.1% | Israel patent granted for REQORSA in combination with PD-1 antibodies. |
| Apr 28 | Preclinical data preview | Positive | -0.5% | Announcement of upcoming positive GPX-002 Type 2 diabetes data at ASGCT. |
| Apr 22 | Clinical biomarker data | Positive | -5.4% | Positive clinical biomarker data for REQORSA to appear at ASCO 2026. |
| Apr 21 | Research agreement | Positive | -5.6% | Sponsored research deal with MD Anderson to study REQORSA biomarkers. |
| Apr 20 | Preclinical cancer data | Positive | -11.2% | Positive preclinical REQORSA lung cancer data presented at AACR 2026. |
Recent positive R&D and IP updates often saw weak or negative next-day price reactions, with only one of five positive events producing an immediate gain.
Over the past month, Genprex has reported multiple positive developments, including a new Israel patent for REQORSA on Apr 30, 2026, preclinical GPX-002 diabetes data for ASGCT on Apr 28, 2026, and encouraging biomarker and trial progress updates for REQORSA in lung cancer. Despite these constructive milestones, four of five prior news events led to negative 24-hour moves, suggesting a pattern where the stock often faded good news. Today’s detailed preclinical diabetes data builds directly on the April ASGCT preview announcement.
Market Pulse Summary
This announcement details positive preclinical results for Genprex’s GPX-002 (Pdx1/MafA) gene therapy, showing reversal of hyperglycemia and improved β-cell function within four weeks in Type 2 diabetic mouse models. It operationalizes an earlier ASGCT preview by providing mechanistic data on insulin secretion, β-cell maturation, and pancreatic delivery. In the broader context of recent REQORSA cancer updates and ongoing losses disclosed in filings, investors may watch for progression toward clinical studies, financing developments, and additional data readouts across both oncology and diabetes programs.
Key Terms
cmv promoter medical
homa-ir medical
endoscopic retrograde cholangiopancreatography medical
AI-generated analysis. Not financial advice.
Pdx1/MafA Gene Therapy Reversed Hyperglycemia in Type 2 Diabetic Models Within Four Weeks
"We are pleased to have our collaborators present this promising preclinical data in Type 2 diabetes at the ASGCT Annual Meeting," said Ryan Confer, President and Chief Executive Officer at Genprex. "We believe this preclinical data is a pivotal step toward a transformative treatment for Type 2 diabetes by directly addressing beta-cell dysfunction. Our therapy holds the potential to offer long-term glycemic control, moving beyond symptomatic management to fundamentally address the disease for diabetic patients."
The featured Genprex-supported abstract and poster at the 2026 ASGCT 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 retrograde infusion into the pancreatic duct 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.
To view the poster presented at the 2026 ASGCT Annual Meeting, please visit Genprex's website here.
About Genprex, Inc.
Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprex's technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. Genprex's oncology program utilizes its systemic, non-viral Oncoprex® Delivery System which encapsulates the gene-expressing plasmids using lipid-based nanoparticles in a lipoplex form. The resultant product is administered intravenously, where it is taken up by tumor cells that then express tumor suppressor proteins that were deficient in the tumor. The Company's lead product candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid), is being evaluated in two clinical trials as a treatment for NSCLC and SCLC. Each of Genprex's lung cancer clinical programs has received a Fast Track Designation from the FDA for the treatment of that patient population, and Genprex's SCLC program has received an FDA Orphan Drug Designation. Genprex's diabetes gene therapy approach is comprised of a novel infusion process that uses an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, GPX-002 transforms 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 a similar approach for Type 2 diabetes, where autoimmunity is not at play, GPX-002 is believed to rejuvenate and replenish exhausted beta cells.
Interested investors and shareholders are encouraged to sign up for press releases and industry updates by visiting the Company Website, registering for Email Alerts and by following Genprex on Twitter, Facebook and LinkedIn.
Cautionary Language Concerning Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made on the basis of the current beliefs, expectations and assumptions of management, are not guarantees of performance and are subject to significant risks and uncertainty. These forward-looking statements should, therefore, be considered in light of various important factors, including those set forth in Genprex's reports that it files from time to time with the Securities and Exchange Commission and which you should review, including those statements under "Item 1A – Risk Factors" in Genprex's Annual Report on Form 10-K for the year ended December 31, 2025.
Because forward-looking statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: Genprex's ability to advance the clinical development, manufacturing and commercialization of its product candidates in accordance with projected timelines and specifications; the timing and success of Genprex's clinical trials, its intended regulatory submissions and any resulting regulatory approvals; the effect of Genprex's product candidates, alone and in combination with other therapies, on cancer and diabetes, including offering a potentially promising avenue for long-term glycemic control in Type 2 diabetes; Genprex's future growth and financial status, including Genprex's ability to maintain compliance with the continued listing requirements of The Nasdaq Capital Market and to continue as a going concern and to obtain capital to meet its long-term liquidity needs on acceptable terms, or at all; Genprex's commercial and strategic partnerships, including those with its third party vendors, suppliers and manufacturers and their ability to successfully perform and scale up the manufacture of its product candidates; Genprex's intellectual property and licenses; and Genprex's current expectations, estimates, forecasts and projections about the industry and markets in which it operates.
These forward-looking statements should not be relied upon as predictions of future events and Genprex cannot assure you that the events or circumstances discussed or reflected in these statements will be achieved or will occur. If such forward-looking statements prove to be inaccurate, the inaccuracy may be material. You should not regard these statements as a representation or warranty by Genprex or any other person that Genprex will achieve its objectives and plans in any specified timeframe, or at all. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Genprex disclaims any obligation to publicly update or release any revisions to these forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this press release or to reflect the occurrence of unanticipated events, except as required by law.
Genprex, Inc.
(877) 774-GNPX (4679)
GNPX Investor Relations
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SOURCE Genprex, Inc.