aibioGenprex reports preclinical diabetes gene therapy data at ASGCT
Tickers GNPX
Research collaborators presented data showing the company's Pdx1 and MafA gene therapy reversed hyperglycemia in a type 2 diabetes mouse model at the 2026 ASGCT Annual Meeting in Boston.
Key facts
- Company
- Genprex, Inc. (NASDAQ: GNPX)
- News type
- Company Updates
- Primary source
- Genprex, Inc. 8-K (2026-05-14)
Genprex, Inc. (Nasdaq: GNPX) issued a press release on May 14, 2026 saying that its research collaborators presented preclinical data on its diabetes gene therapy drug candidate at the 2026 American Society of Gene and Cell Therapy Annual Meeting on May 13, 2026 in Boston, Massachusetts. The candidate is also known as PM or GPX-002. The presented abstract and poster is titled "Pancreatic Delivery of AAV-Pdx1/MafA Reverses Hyperglycemia in a Preclinical Model of Type 2 Diabetes." The company disclosed the release in a Form 8-K filed the same day.
The study design
Eight-week-old male C57BL/6 mice were kept on a regular diet or a high fat diet for 24 weeks. The high fat diet mice then either remained unoperated or underwent retrograde infusion into the pancreatic duct. The infusion used adeno-associated virus AAV-8 encoding Pdx1 and MafA cassettes under either the CMV promoter, which targets islet cells globally, or the rat insulin promoter, which targets beta cells specifically. A control virus group was also included. The diet did not change after surgery.
At two and or four weeks after surgery, researchers ran glucose tolerance tests, insulin tolerance tests, glucose-stimulated insulin secretion tests, and HOMA-IR calculations, and they assessed glucagon secretion. Mice were then euthanized for pancreatic histology, measurement of beta and alpha cell mass, electron microscopy, and single-cell RNA sequencing of isolated islets.
The results at four weeks
The filing reports major improvements in the control of diabetes at four weeks. Islets from high fat diet mice treated with the CMV-PM-GFP construct secreted insulin at levels similar to islets from regular diet mice, and both groups secreted more than the control high fat diet groups. The rat insulin promoter construct, which targets beta cells, reversed hyperglycemia in the high fat diet mice and improved ex vivo insulin secretion.
Electron microscopy showed that PM treatment increased the number of total and mature insulin granules and decreased immature granules compared with high fat diet controls. Transcriptomic pseudotime analysis showed beta cells shifting from an immature state toward a more mature state after PM treatment. The company said the results suggest PM gene therapy reverses hyperglycemia, likely in large part by enhancing beta cell function and maturation.
The 10-K background and finances
The company said the approach is technically translatable to humans using endoscopic retrograde cholangiopancreatography to deliver the gene therapy to the pancreas. The company believes the preclinical data is a pivotal step toward a potentially transformative treatment for type 2 diabetes, and that the therapy may offer long-term glycemic control.
The 10-K business section describes the company's lead oncology work with REQORSA, including the Acclaim-1 trial in lung cancer. The 10-K says Acclaim-1 is enrolling in its Phase 2a expansion portion, and that the company expects an interim analysis in the second half of 2026.
For the fiscal year ended December 31, 2025, the XBRL data show research and development expense of $9.4 million and a net loss of $16.2 million. For 2024, the comparable figures were $10.5 million and $21.1 million. Cash from operations was negative $15.3 million in 2025 and negative $17.9 million in 2024. Year-end cash and cash equivalents for 2024 were $1.6 million.
What comes next
The company said the therapy holds the potential to move beyond symptomatic management to fundamentally address the disease for diabetic patients. The filing does not give dates for further preclinical or clinical work on the diabetes candidate.
The evidence has hard limits. It comes from male mice only, on a high fat diet, and the company calls the data positive but the filing reports no animal numbers, p-values or effect sizes, and nothing on durability beyond four weeks. A mouse reversal of hyperglycemia is not a result in people, and the filing reports no safety data for AAV infusion into the pancreatic duct, a route the company says could be done in humans with endoscopic retrograde cholangiopancreatography. Calling the data a pivotal step is the company's phrase, and the filing reports no regulatory milestone. The company reported no 2024 revenue and a 2025 net loss of $16.2 million.