Genprex Collaborators to Present Positive Preclinical Data on Diabetes Gene Therapy at the ASGCT 28th Annual Meeting
Genprex announced that its research collaborators will present positive preclinical data for their diabetes gene therapy drug candidate GPX-002 at the ASGCT 28th Annual Meeting in New Orleans this May.
The research focuses on a novel gene therapy using recombinant adeno-associated virus (rAAV) to treat diabetes mellitus. Key findings show that retrograde intraductal infusion of rAAV6 successfully delivered Pdx1 and MafA genes, converting alpha cells into insulin-secreting beta-like cells.
Testing on non-human primates demonstrated:
- Improved glucose tolerance and reduced insulin needs one month post-infusion
- Durable effects using AAV6 capsid with endocrine-specific promoters
- Successful immune response management using temporary immunosuppression
- Sustained therapeutic effects even after stopping immunosuppression
The research represents a potential breakthrough in diabetes treatment, with results showing sustained therapeutic effects without long-term immunosuppression requirements.
Genprex ha annunciato che i suoi collaboratori di ricerca presenteranno dati preclinici positivi per il loro candidato farmaco per la terapia genica del diabete, GPX-002, al 28° Congresso Annuale ASGCT che si terrà a New Orleans questo maggio.
La ricerca si concentra su una nuova terapia genica che utilizza il virus adeno-associato ricombinante (rAAV) per il trattamento del diabete mellito. I risultati chiave mostrano che l'infusione retrograda intraduttale di rAAV6 ha efficacemente trasportato i geni Pdx1 e MafA, convertendo le cellule alfa in cellule simili a beta che secernono insulina.
I test su primati non umani hanno dimostrato:
- Miglioramento della tolleranza al glucosio e riduzione del fabbisogno di insulina a un mese dall'infusione
- Effetti duraturi grazie all'uso del capside AAV6 con promotori specifici per le cellule endocrine
- Gestione efficace della risposta immunitaria mediante immunosoppressione temporanea
- Effetti terapeutici sostenuti anche dopo la sospensione dell'immunosoppressione
Questa ricerca rappresenta una possibile svolta nel trattamento del diabete, con risultati che indicano effetti terapeutici duraturi senza la necessità di immunosoppressione a lungo termine.
Genprex anunció que sus colaboradores de investigación presentarán datos preclínicos positivos para su candidato a medicamento de terapia génica para la diabetes, GPX-002, en la 28ª Reunión Anual de ASGCT en Nueva Orleans este mayo.
La investigación se centra en una novedosa terapia génica que utiliza virus adenoasociado recombinante (rAAV) para tratar la diabetes mellitus. Los hallazgos clave muestran que la infusión intraductal retrógrada de rAAV6 entregó con éxito los genes Pdx1 y MafA, convirtiendo las células alfa en células similares a beta que secretan insulina.
Las pruebas en primates no humanos demostraron:
- Mejora en la tolerancia a la glucosa y reducción de la necesidad de insulina un mes después de la infusión
- Efectos duraderos utilizando la cápside AAV6 con promotores específicos endocrinos
- Gestión exitosa de la respuesta inmune mediante inmunosupresión temporal
- Efectos terapéuticos sostenidos incluso después de suspender la inmunosupresión
La investigación representa un posible avance en el tratamiento de la diabetes, con resultados que muestran efectos terapéuticos sostenidos sin necesidad de inmunosupresión a largo plazo.
Genprex는 연구 협력자들이 이번 5월 뉴올리언스에서 열리는 ASGCT 제28차 연례회의에서 당뇨병 유전자 치료 후보 약물 GPX-002의 긍정적인 전임상 데이터를 발표할 것이라고 밝혔습니다.
연구는 재조합 아데노연관바이러스(rAAV)를 이용한 새로운 유전자 치료법에 초점을 맞추고 있으며, 주요 결과는 rAAV6의 역행성 관내 주입이 Pdx1과 MafA 유전자를 성공적으로 전달하여 알파 세포를 인슐린을 분비하는 베타 유사 세포로 전환시켰음을 보여줍니다.
비인간 영장류 실험 결과:
- 주입 1개월 후 포도당 내성 개선 및 인슐린 필요량 감소
- 내분비 특이 프로모터를 가진 AAV6 캡시드를 사용한 지속적인 효과
- 일시적 면역억제를 통한 면역 반응 성공적 관리
- 면역억제 중단 후에도 지속되는 치료 효과
이 연구는 장기 면역억제 없이 지속적인 치료 효과를 보이는 당뇨병 치료의 잠재적 돌파구를 제시합니다.
Genprex a annoncé que ses collaborateurs de recherche présenteront des données précliniques positives pour leur candidat médicament de thérapie génique contre le diabète, GPX-002, lors de la 28e réunion annuelle de l'ASGCT à la Nouvelle-Orléans en mai prochain.
La recherche porte sur une nouvelle thérapie génique utilisant un virus adéno-associé recombinant (rAAV) pour traiter le diabète sucré. Les résultats clés montrent que l'infusion intraductale rétrograde de rAAV6 a permis de délivrer avec succès les gènes Pdx1 et MafA, transformant les cellules alpha en cellules de type bêta sécrétrices d'insuline.
Les tests sur des primates non humains ont démontré :
- Une amélioration de la tolérance au glucose et une réduction des besoins en insuline un mois après l'infusion
- Des effets durables grâce à l'utilisation de la capside AAV6 avec des promoteurs spécifiques aux cellules endocrines
- Une gestion efficace de la réponse immunitaire grâce à une immunosuppression temporaire
- Des effets thérapeutiques soutenus même après l'arrêt de l'immunosuppression
Cette recherche représente une avancée potentielle dans le traitement du diabète, avec des résultats montrant des effets thérapeutiques durables sans nécessiter d'immunosuppression à long terme.
Genprex gab bekannt, dass seine Forschungspartner positive präklinische Daten für ihren Gentherapie-Wirkstoffkandidaten GPX-002 zur Behandlung von Diabetes mellitus auf der 28. Jahrestagung der ASGCT im Mai in New Orleans präsentieren werden.
Die Forschung konzentriert sich auf eine neuartige Gentherapie mit rekombinantem adeno-assoziiertem Virus (rAAV) zur Behandlung von Diabetes mellitus. Zentrale Ergebnisse zeigen, dass die retrograde intraduktale Infusion von rAAV6 die Gene Pdx1 und MafA erfolgreich übermittelte und Alphazellen in insulinproduzierende Beta-ähnliche Zellen umwandelte.
Tests an nicht-menschlichen Primaten zeigten:
- Verbesserte Glukosetoleranz und reduzierter Insulinbedarf einen Monat nach der Infusion
- Nachhaltige Effekte durch Verwendung der AAV6-Kapsid mit endokrinspezifischen Promotoren
- Erfolgreiches Immunmanagement durch temporäre Immunsuppression
- Anhaltende therapeutische Effekte auch nach Absetzen der Immunsuppression
Die Forschung stellt einen potenziellen Durchbruch in der Diabetesbehandlung dar, da die Ergebnisse nachhaltige therapeutische Effekte ohne langfristige Immunsuppression zeigen.
- Positive preclinical data for GPX-002 diabetes gene therapy showing improved glucose tolerance in non-human primates
- Selection to present at ASGCT's Annual Meeting validates the diabetes program's potential
- Demonstrated durable therapeutic effects using AAV6 capsid with endocrine-specific promoters
- Successful immune response management achieved through temporary immunosuppression
- Sustained therapeutic effects observed even after discontinuation of immunosuppression
- Still in preclinical stage, indicating long pathway to potential commercialization
- Requires immunosuppression therapy for effectiveness, which could limit patient eligibility
- Potential immune response challenges to AAV delivery system
Insights
Genprex's gene therapy shows promising diabetes treatment results in non-human primates; immunosuppression protocol effectively prevents anti-viral immunity while maintaining therapeutic benefits.
Genprex's GPX-002 represents a novel approach to diabetes treatment using recombinant adeno-associated virus (rAAV) to deliver Pdx1 and MafA genes. This clever genetic reprogramming converts pancreatic alpha cells into insulin-secreting beta-like cells, addressing the fundamental pathology of diabetes - the lack of functional insulin-producing cells.
The non-human primate (NHP) data demonstrates encouraging efficacy markers, with subjects showing improved glucose tolerance and reduced insulin requirements just one month post-infusion. Particularly noteworthy is their solution to the immune response challenge that frequently derails gene therapies. Their temporary immunosuppression protocol using rituximab, rapamycin, and steroids effectively prevented anti-viral immunity while allowing therapeutic effects to persist even after discontinuing immunosuppression.
Selection for presentation at ASGCT, one of gene therapy's premier scientific forums, provides external validation of the approach's scientific merit. However, we must maintain perspective - these remain preclinical results with significant translational challenges ahead, including safety profile verification, dosing optimization, and durability assessments before human applications can advance.
The research effectively addresses two critical hurdles in diabetes gene therapy: functional reprogramming of native cells and mitigation of immune responses to viral vectors. This represents meaningful scientific progress in a challenging therapeutic space.
Genprex's diabetes gene therapy shows promising preclinical results; selection for prestigious ASGCT conference validates approach but commercialization remains years away.
Genprex's gene therapy approach for diabetes targets an enormous market opportunity with substantial unmet need. The therapeutic concept - potentially enabling a one-time treatment versus lifetime insulin dependency - represents the kind of paradigm-shifting approach that could create significant value if successfully developed.
The preclinical non-human primate data provides early validation of two critical components: the therapy's mechanism of action and the company's approach to overcoming immune response challenges. Their temporary immunosuppression protocol shows particular promise in addressing a major hurdle that has limited gene therapy applications broadly.
Selection for presentation at ASGCT, the premier gene therapy scientific conference, delivers increased visibility within the specialized gene therapy community and potentially enhances partnership opportunities. For Genprex specifically, this diabetes program diversifies their pipeline beyond oncology, potentially creating multiple value-generating pathways and reducing overall company risk.
However, investors should maintain appropriate perspective - while promising, GPX-002 remains years from potential commercialization with significant developmental and regulatory hurdles ahead. The competitive landscape for innovative diabetes treatments is intensely crowded with multiple therapeutic approaches being pursued by well-capitalized competitors. Nevertheless, this preclinical progress represents a positive signal regarding the scientific validity of Genprex's approach in a disease area affecting hundreds of millions worldwide.
Novel Gene Therapy Program Offers a Promising Opportunity for Curative Therapy in Diabetes
"We are proud of the preclinical data supporting our novel gene therapy program for diabetes, and we believe selection to present our program at one of the premier events in cell and gene therapy provides another point of validation for our diabetes program," said Ryan Confer, President and Chief Executive Officer at Genprex. "We look forward to sharing our GPX-002 data with industry leaders engaged in advancing the latest scientific research and new technologies."
The oral presentation details for the Genprex-supported abstract:
Abstract Title: Immune Modulation Sustains Alpha Cell Reprogramming and Mitigates Immune Responses to AAV in a Diabetic Non-Human Primate Model
Session Title: Challenges in Immunological Responses to Therapeutic Interventions
Presenter: Hannah Rinehardt, MD, University of Pittsburgh Medical Center
Presentation Date: May 16, 2025
Presentation Time: 4:15 – 4:30 p.m. CT
Location: Room 278-282
The featured Genprex-supported abstract to be presented for oral presentation at the ASGCT 28th Annual Meeting:
Gene therapy using recombinant adeno-associated virus (rAAV) offers a promising opportunity for curative therapy in diabetes mellitus. Retrograde intraductal infusion of rAAV6 to deliver Pdx1 and MafA, converting alpha cells into beta-like cells that secrete insulin physiologically, reverses diabetes in mouse models. Little is known about the direct infusion of AAV into the pancreatic duct for gene therapy in non-human primates (NHPs). In clinical trials, anti-viral immunity to AAV can be a barrier to successful gene therapy. Researchers evaluated the immune response to direct infusion of rAAV into the pancreatic duct of NHPs with streptozotocin-induced diabetes and evaluated how to best manage immune responses.
Diabetes was induced with streptozotocin (STZ) in cynomolgus macaques. 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. Blood work including serum C peptide and IV glucose tolerance tests were serially obtained to monitor therapeutic efficacy. Immune response monitoring was performed for up to 4 months post-infusion and included serial NAbs, ELISpot assays, and immunophenotyping. Pancreatic tissues were analyzed using IHC and RNA-scope for beta cell markers, as well as single-cell RNA transcriptomics.
One-month post-infusion, NHPs showed improved glucose tolerance and reduced insulin requirements. The AAV6 capsid with endocrine-specific promoters driving Pdx1 and MafA showed durable effects. ELISpot-positive cytotoxic T cells and neutralizing antibodies developed when steroids were absent. With steroid-sparing regimens, pancreatic B and T lymphocyte populations were noted on scRNA 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. Despite complete IS discontinuation at 3 months post-infusion, meaningful anti-viral immune response was not mounted up to one month after IS withdrawal as evidenced by low NAb titers and negative ELISpot analysis. Additionally, rAAV gene therapy in these animals remained effective and glucose tolerance continued improving in the absence of immunosuppression.
In conclusion, researchers developed a novel rAAV gene therapy approach and demonstrated that infusion of rAAV directly into the pancreatic duct of NHPs induces an anti-viral immune response. The anti-viral immune response in NHPs can be largely prevented by administration of a multi-agent IS and can allow for sustained therapeutic effects.
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.
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, 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.
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