Genprex Collaborators Present Research on Non-Viral Approach to Diabetes Gene Therapy Using Lipid Nanoparticle Delivery System at the 2025 American Diabetes Association 85th Scientific Sessions

Rhea-AI Summary

Genprex (NASDAQ: GNPX) presented positive preclinical data for GPX-002, their diabetes gene therapy candidate, at the 2025 ADA Scientific Sessions. The research showcased a novel non-viral lipid nanoparticle (LNP) delivery system for diabetes gene therapy, demonstrating successful transfection of pancreatic cells through biliary duct infusion. Two specific LNPs showed high efficiency in transfecting α- and β-cells in mouse Islets of Langerhans, with the ALX-184 LNP achieving 100 times more luciferase activity in the pancreas compared to other organs. This breakthrough approach could enable repeat dosing without requiring immunosuppression, potentially offering advantages over traditional AAV delivery methods in treating Type 1 Diabetes.

Positive

• Novel non-viral delivery system shows potential for repeat dosing in diabetes treatment
• Selected LNP demonstrated 100x more luciferase activity in pancreas vs other organs, showing high specificity
• Successful transfection of pancreatic cells achieved without need for immunosuppression
• Research represents first-of-its-kind approach using LNP in pancreatic duct instead of viral AAV construct

Negative

• Research still in early preclinical stage
• Additional studies needed to validate approach
• Multiple delivery systems being explored simultaneously (AAV and LNP) could divide resources

Insights

Biotechnology Researcher

Genprex shows promising preclinical results using lipid nanoparticles to deliver diabetes gene therapy, potentially enabling repeat dosing.

Genprex has presented an exciting advancement in their diabetes gene therapy program by developing a non-viral lipid nanoparticle (LNP) delivery system for their GPX-002 candidate. This represents a significant technical innovation compared to traditional viral vectors like AAVs used in gene therapy.

The key breakthrough here is the demonstration that specific LNPs can efficiently transfect α- and β-cells in the islets of Langerhans when delivered through the biliary/pancreatic duct. One particular formulation using ALX-184 showed remarkable pancreatic specificity, with 100 times more activity in the pancreas than other organs. This high degree of targeting is crucial for minimizing off-target effects.

The most significant potential advantage of this approach is enabling repeat dosing, which isn't possible with AAV vectors due to the immune response they generate. For diabetes treatment, this could be particularly valuable as it might allow for dosage adjustments or maintenance therapy over time. Additionally, the approach may eliminate the need for immunosuppression normally required with viral vectors.

While these results are promising, it's important to note this research remains preclinical, using mouse models rather than human subjects. The company is pursuing this alongside their existing AAV-based approach as a potential next-generation optimization. The ability to deliver therapeutic genes specifically to pancreatic islet cells without viral vectors could substantially improve the treatment paradigm for Type 1 diabetes if successfully translated to humans.

Novel Diabetes Gene Therapy Shows Potential for Re-Dosing Using Non-Viral Delivery System

AUSTIN, Texas, June 23, 2025 /PRNewswire/ -- 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, today announced that its research collaborators presented positive preclinical data and research from studies of GPX-002, the Company's diabetes gene therapy drug candidate, at the 2025 American Diabetes Association (ADA) 85^th Scientific Session in Chicago. These studies used an alternative second-generation approach with a non-viral lipid nanoparticle delivery system. This exploratory research represents the Company's forward-thinking work within its diabetes program to determine if the novel diabetes gene therapy can be delivered using a non-viral delivery system which could allow for repeat dosing.

"This is a huge step forward for our research that demonstrates our ability to use a lipid nanoparticle with a nucleic acid payload to transfect cells within the islets of Langerhans via a direct infusion into the biliary duct/pancreatic duct," said Ryan Confer, President and Chief Executive Officer at Genprex. "We believe we are the first and only company to be doing this type of research in the pancreatic duct using a lipid nanoparticle in place of a viral AAV construct. These findings provide the proof of concept needed to move forward with additional studies and opens the door for a next generation approach of our novel gene therapy that would allow repeat dosing of patients, which may prove to be very important in the potential treatment of Type 1 Diabetes."

The featured Genprex-supported abstract presented at the ADA 85^th Scientific Sessions:

Title: Selecting Lipid Nanoparticles for Transfection of Islets of Langerhans Cells
Type: Poster Presentation
Category: 20 Beta-Cell Replacement
Poster Number: 1567-B
Presentation Date: Saturday, June 21, 2025
Presentation Time: 12:30 – 1:30 p.m. CT
Presenter: József Répási, AldexChem LTD

The poster is available on Genprex's website.

In this study, nine lipid nanoparticles (LNPs) prepared with patented LipexSil^® lipids with a green fluorescent protein (GFP) messenger RNA (mRNA) payload were evaluated for transfection efficiency in isolated mouse Islets of Langerhans. Two specific LNPs were highly efficient in transfecting α- and β-cells in isolated mouse Islets of Langerhans. LNP made using ALX-184 with a Luciferase mRNA payload injected into the mouse common bile duct efficiently transfected pancreatic cells, and thus efficiently crossed the basement membrane separating the pancreatic duct from pancreatic cells. The same LNP made using ALX-184 with a GFP mRNA payload injected into the mouse common bile duct also efficiently transfected mouse Islet of Langerhans cells.

"We are proud of this groundbreaking research in diabetes using LNP prepared with a patented lipid, which initially evaluated transfection efficiency in isolated mouse islets of Langerhans and showed that the transfection was highly efficient," said Mark Berger, MD, Chief Medical Officer at Genprex. "We tested several lipids and selected one that was less toxic than a lipid that is commercially used in vaccines. Using our selected LNP with a luciferase mRNA payload, there was 100 times more luciferase activity in the pancreas than in other organs, demonstrating the specificity of transduction using infusion into the biliary duct/pancreatic duct. We were then also able to demonstrate the ability of this LNP to cross the basement membrane in the pancreatic duct and to penetrate into the islets of Langerhans in the pancreas and transfect cells there. Overall, these exciting data pave the way for additional studies that could improve on today's treatments for diabetes."

Genprex's research with its contract development and manufacturing organization partner is in addition to its ongoing preclinical studies using an AAV construct. This new research is focused on evaluating potential next generation construct optimization. While AAV delivery is a well understood delivery mechanism, there could be many benefits to a non-viral delivery system, including the potential for re-dosing patients to optimize treatment. Additionally, there may be no need for immunosuppression since there is no immune response against LNPs.

About GPX-002
GPX-002, which has been exclusively licensed from the University of Pittsburgh, is currently being developed using the same construct for the treatment of both Type 1 diabetes (T1D) and Type 2 diabetes (T2D). The same general novel approach is used in each of T1D and T2D whereby an adeno-associated virus (AAV) vector containing the Pdx1 and MafA genes is administered directly into the pancreatic duct. In humans, this can be done with a routine endoscopy procedure. In T1D, GPX-002 is designed to work by transforming 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 vivo, preclinical studies show that GPX-002 restored normal blood glucose levels for an extended period of time in T1D mouse models. In T2D, where autoimmunity is not at play, GPX-002 is believed to rejuvenate and replenish exhausted beta cells. The pioneering research discussed above is aimed at developing an alternative delivery system for this transdifferentiation therapy that can be administered more than once.

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, 2024.

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 and regulatory approvals, including but not limited to, the Company's beliefs about the anticipated effects of GPX-002 and its potential as a therapeutic approach, whether using AAV delivery or a potential second generation non-viral delivery system; the effect of Genprex's product candidates, alone and in combination with other therapies, on cancer and diabetes; the effects of any strategic research and development prioritization initiatives, and any other strategic alternatives or other efforts that Genprex takes or may take in the future that are aimed at optimizing and re-focusing Genprex's diabetes, oncology and/or other clinical development programs including prioritization of resources, and the extent to which Genprex is able to implement such efforts and initiatives successfully to achieve the desired and intended results thereof; 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; and Genprex's intellectual property and licenses.

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)

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FAQ

What is the significance of Genprex's GPX-002 diabetes gene therapy research presented at ADA 2025?

The research demonstrates a breakthrough non-viral delivery system using lipid nanoparticles that could enable repeat dosing of gene therapy for diabetes treatment without requiring immunosuppression.

How effective is GNPX's new lipid nanoparticle delivery system for diabetes gene therapy?

The selected ALX-184 LNP showed high efficiency, achieving 100 times more luciferase activity in the pancreas compared to other organs, with successful transfection of α- and β-cells in mouse Islets of Langerhans.

What advantages does Genprex's non-viral delivery system offer over traditional methods?

The non-viral LNP delivery system allows for potential repeat dosing and eliminates the need for immunosuppression, as there is no immune response against LNPs, unlike traditional AAV delivery methods.

What stage of development is Genprex's GPX-002 diabetes gene therapy in?

GPX-002 is currently in preclinical development, with ongoing studies using both AAV construct and the new non-viral LNP delivery system.

How does GNPX's lipid nanoparticle delivery system work for diabetes treatment?

The system delivers genetic material via lipid nanoparticles through direct infusion into the biliary duct/pancreatic duct, efficiently transfecting cells within the islets of Langerhans.