Genprex Collaborators Present Positive Preclinical Research on Diabetes Gene Therapy at the 2025 American Diabetes Association 85th Scientific Sessions
Genprex (NASDAQ: GNPX) presented positive preclinical research results for GPX-002, its diabetes gene therapy drug candidate, at the 2025 American Diabetes Association Scientific Sessions. The therapy demonstrated promising results in non-human primates (NHPs) with streptozotocin-induced diabetes.
The research showed that GPX-002, which uses recombinant adeno-associated virus (rAAV) to deliver Pdx1 and MafA genes, successfully converted alpha cells into insulin-secreting beta-like cells. The treated NHPs showed improved glucose tolerance and reduced insulin requirements one month post-infusion. Temporary immunosuppression for 3 months proved effective in preventing anti-viral immunity, with researchers now evaluating a 6-month immunosuppression period.
Genprex (NASDAQ: GNPX) ha presentato risultati preclinici positivi per GPX-002, il suo candidato farmaco per la terapia genica del diabete, durante le Sessioni Scientifiche 2025 dell'American Diabetes Association. La terapia ha mostrato risultati promettenti in primati non umani (NHP) con diabete indotto da streptozotocina.
Lo studio ha dimostrato che GPX-002, che utilizza un virus adeno-associato ricombinante (rAAV) per veicolare i geni Pdx1 e MafA, è riuscito a convertire le cellule alfa in cellule simili a beta produttrici di insulina. I primati trattati hanno evidenziato un miglioramento della tolleranza al glucosio e una riduzione del fabbisogno insulinico a un mese dall'infusione. Un'immunosoppressione temporanea di 3 mesi si è rivelata efficace nel prevenire l'immunità antivirale, mentre i ricercatori stanno ora valutando un periodo di immunosoppressione di 6 mesi.
Genprex (NASDAQ: GNPX) presentó resultados preclínicos positivos para GPX-002, su candidato a terapia génica para la diabetes, en las Sesiones Científicas 2025 de la American Diabetes Association. La terapia mostró resultados prometedores en primates no humanos (NHP) con diabetes inducida por estreptozotocina.
La investigación demostró que GPX-002, que utiliza un virus adenoasociado recombinante (rAAV) para entregar los genes Pdx1 y MafA, logró convertir células alfa en células similares a beta que secretan insulina. Los NHP tratados mostraron mejora en la tolerancia a la glucosa y reducción en los requerimientos de insulina un mes después de la infusión. La inmunosupresión temporal de 3 meses fue efectiva para prevenir la inmunidad antiviral, y los investigadores están evaluando ahora un periodo de inmunosupresión de 6 meses.
Genprex (NASDAQ: GNPX)는 2025년 미국 당뇨병 학회 과학 세션에서 당뇨병 유전자 치료 후보 약물인 GPX-002의 긍정적인 전임상 연구 결과를 발표했습니다. 이 치료법은 스트렙토조토신으로 유도된 당뇨병을 가진 비인간 영장류(NHPs)에서 유망한 결과를 보였습니다.
연구에 따르면 GPX-002는 재조합 아데노연관바이러스(rAAV)를 사용해 Pdx1 및 MafA 유전자를 전달하여 알파 세포를 인슐린 분비 베타 유사 세포로 성공적으로 전환시켰습니다. 치료받은 NHP들은 주입 후 한 달 만에 포도당 내성 개선과 인슐린 요구량 감소를 나타냈습니다. 3개월간의 일시적 면역억제는 항바이러스 면역을 예방하는 데 효과적이었으며, 연구진은 현재 6개월 면역억제 기간을 평가 중입니다.
Genprex (NASDAQ : GNPX) a présenté des résultats précliniques positifs pour GPX-002, son candidat médicament de thérapie génique contre le diabète, lors des sessions scientifiques 2025 de l'American Diabetes Association. La thérapie a montré des résultats prometteurs chez des primates non humains (NHP) atteints de diabète induit par la streptozotocine.
Les recherches ont démontré que GPX-002, qui utilise un virus adéno-associé recombinant (rAAV) pour délivrer les gènes Pdx1 et MafA, a réussi à convertir les cellules alpha en cellules similaires aux cellules bêta sécrétrices d'insuline. Les NHP traités ont présenté une amélioration de la tolérance au glucose et une réduction des besoins en insuline un mois après l'infusion. Une immunosuppression temporaire de 3 mois s'est avérée efficace pour prévenir l'immunité antivirale, les chercheurs évaluant désormais une période d'immunosuppression de 6 mois.
Genprex (NASDAQ: GNPX) präsentierte positive präklinische Forschungsergebnisse für GPX-002, seinen Gentherapie-Kandidaten gegen Diabetes, auf den Wissenschaftlichen Sitzungen der American Diabetes Association 2025. Die Therapie zeigte vielversprechende Ergebnisse bei nicht-menschlichen Primaten (NHPs) mit streptozotocin-induziertem Diabetes.
Die Forschung zeigte, dass GPX-002, das rekombinantes adeno-assoziiertes Virus (rAAV) zur Übertragung der Gene Pdx1 und MafA verwendet, erfolgreich Alphazellen in insulinsekretierende Beta-ähnliche Zellen umwandelte. Die behandelten NHPs zeigten verbesserte Glukosetoleranz und reduzierten Insulinbedarf einen Monat nach der Infusion. Eine vorübergehende Immunsuppression über 3 Monate erwies sich als wirksam zur Verhinderung der antiviralen Immunität, wobei Forscher nun eine 6-monatige Immunsuppression evaluieren.
- Successful conversion of alpha cells to insulin-secreting beta-like cells in non-human primates
- Improved glucose tolerance and reduced insulin requirements observed after one month
- Therapy shows potential for treating both Type 1 and Type 2 diabetes
- Promising results in maintaining glucose homeostasis
- Immunosuppression required for several months to prevent anti-viral immunity
- Discontinuation of immunosuppression at 3 months led to immune response
- Additional studies needed before moving to human clinical trials
- Complex delivery method requiring retrograde intraductal infusion via laparotomy
Insights
Genprex's diabetes gene therapy shows promising preclinical results in non-human primates, with significant implications for diabetes treatment development.
Genprex has presented encouraging preclinical research for their diabetes gene therapy candidate GPX-002 at the American Diabetes Association Scientific Sessions. The therapy uses a gene delivery approach that successfully reprograms pancreatic alpha cells into insulin-producing beta-like cells, showing promising efficacy in non-human primates (NHPs).
The data demonstrates that one month post-infusion, treated NHPs showed improved glucose tolerance and reduced insulin requirements - critical metrics for diabetes therapy efficacy. The mechanism involves delivering Pdx1 and MafA genes via adeno-associated virus (rAAV) directly into the pancreatic duct, triggering cell transdifferentiation without requiring conventional immunosuppression for the therapy itself.
What's particularly significant is the colocalization of insulin and glucagon in treated islets, indicating successful transdifferentiation of alpha cells to beta-like cells that retain some original functionality while gaining insulin-producing capabilities. This dual-hormone potential represents a sophisticated approach to glucose regulation.
The research identified important clinical development considerations, particularly that temporary immunosuppression is necessary to prevent immune responses against the viral vector proteins. Three-month immunosuppression regimens showed efficacy but immune responses occurred after discontinuation, suggesting longer immunosuppression periods may be needed for sustained therapeutic effect.
For Genprex, these results validate their gene therapy approach and move them closer to human clinical trials. The ongoing studies in both Type 1 and Type 2 diabetes NHP models will generate critical data to support eventual clinical development, particularly regarding optimal immunosuppression protocols and long-term efficacy.
Latest Research Demonstrates Promising Improved Glucose Homeostasis by Reprogramming Alpha Cells
"We are proud of the research presented at the ADA conference, which demonstrates that alpha cells in animal models of T1D have undergone transdifferentiation to beta-like cells after being transduced with GPX-002. In addition, the beta-like cells were still providing improved control of glucose levels after three months," said Ryan Confer, President and Chief Executive Officer at Genprex. "We are very excited to continue evaluating GPX-002 as we further optimize how to control anti-viral immunity and move toward human clinical trials."
The oral presentation details for the Genprex-supported abstract at the ADA 85th Scientific Sessions:
Title: Recombinant AAV-mediated Gene Therapy For The Treatment Of Streptozotocin-Induced Diabetes in Non-Human Primates
Type: Oral Presentation
Track: Immunology/Beta-Cell Replacement
Category: Exploring Alternative Strategies to Stem Cell-Derived Beta Cells and Insights from Autologous Islet Transplantation
Presentation Date: Monday, June 23, 2025
Presentation Time: 1:30 p.m. CT
Presenter: Hannah Rinehardt, MD, University of Pittsburgh Medical Center
The oral presentation discussed GPX-002, the diabetes gene therapy, which uses infusion of recombinant adeno-associated virus (rAAV) retrograde into the pancreatic duct to deliver the Pdx1 and MafA genes. The gene therapy converts alpha cells into beta-like cells that secrete insulin physiologically, reversing diabetes in mouse models, where immunosuppression was not necessary. Researchers evaluated the immune response to direct infusion of rAAV into the pancreatic duct of non-human primates (NHPs) with streptozotocin-induced diabetes and evaluated how to best manage immune responses against the virus capsid proteins.
Diabetes was induced with streptozotocin (STZ) in cynomolgus macaques, a type of NHP. NHPs received retrograde intraductal infusion of rAAV via laparotomy for precise delivery to the pancreas. rAAV capsids were chosen based on tropism for endocrine cells, and pre-existing neutralizing antibody titers (NAbs) were negative.
Expression of viral proteins occurs for a limited period of time after rAAV infection, since the infection doesn't produce new AAV virus. One-month post-infusion, NHPs showed improved glucose tolerance and reduced insulin requirements. In the following months, using steroid-sparing regimens, increases in pancreatic B and T lymphocyte populations were noted on single cell RNA sequencing. Temporary immunosuppression (IS), using a combination of rituximab, rapamycin, and steroids for a 3-month course is largely effective at preventing anti-viral immunity. However, discontinuation of IS at 3 months post-infusion led to an immune response afterwards, indicating that IS in NHPs may need to be continued longer, and six months of IS in NHPs is now being evaluated.
When colocalization of insulin and glucagon is quantified, there was significantly elevated colocalization in treated islets compared to untreated diabetic and non-diabetic controls. This suggests that GPX-002 can lead to transdifferentiation of alpha cells into a new population of beta-like cells that make insulin but still retain the capacity to produce glucagon.
In conclusion, the novel rAAV gene therapy research demonstrated that infusion of rAAV directly into the pancreatic duct of NHPs leads to transdifferentiation of alpha cells to beta-like cells with restoration of glucose homeostasis. IS, including steroids, is necessary for a number of months to prevent anti-viral immunity in NHPs.
Researchers are continuing preclinical studies of GPX-002 therapy in NHP models of both Type 1 and Type 2 diabetes to generate additional data, and current studies are evaluating viral efficacy after six months of IS.
About GPX-002
GPX-002, which has been exclusively licensed from the University of
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.
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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; 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.
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