Genprex Advances Diabetes Program with Addition of Research Focused on Non-Viral Delivery System
Genprex (NASDAQ: GNPX) announced advancement in its diabetes program through a strategic collaboration with a CDMO partner to research a non-viral lipid nanoparticle delivery system for its diabetes gene therapy drug candidate. This alternative approach represents a second-generation development, separate from existing preclinical programs.
The company's current diabetes treatment, GPX-002, uses an AAV vector containing Pdx1 and MafA genes administered into the pancreatic duct. While effective, viral delivery systems cannot be readministered due to immune responses. The non-viral approach could enable multiple treatments, potentially optimizing patient outcomes.
Recent preclinical studies showed GPX-002's effectiveness in Type 1 NOD mouse models, maintaining glucose control for approximately 120 days, significantly longer than the 17-day median observed with traditional islet cell transplants. The therapy is being developed for both Type 1 and Type 2 diabetes, working by transforming pancreatic alpha cells into functional beta-like cells.
Genprex (NASDAQ: GNPX) ha annunciato progressi nel suo programma per il diabete attraverso una collaborazione strategica con un partner CDMO per ricercare un sistema di consegna non virale a base di nanoparticelle lipidiche per il suo candidato farmaco per la terapia genica del diabete. Questo approccio alternativo rappresenta uno sviluppo di seconda generazione, distinto dai programmi preclinici esistenti.
Il trattamento attuale dell'azienda per il diabete, GPX-002, utilizza un vettore AAV contenente i geni Pdx1 e MafA somministrato nel dotto pancreatico. Sebbene efficace, i sistemi di somministrazione virale non possono essere ri-somministrati a causa delle risposte immunitarie. L'approccio non virale potrebbe consentire più trattamenti, ottimizzando potenzialmente i risultati per i pazienti.
Recenti studi preclinici hanno dimostrato l'efficacia di GPX-002 nei modelli murini NOD di Tipo 1, mantenendo il controllo della glicemia per circa 120 giorni, significativamente più a lungo rispetto alla media di 17 giorni osservata con i tradizionali trapianti di cellule isletiche. La terapia è in fase di sviluppo sia per il diabete di Tipo 1 che di Tipo 2, lavorando per trasformare le cellule alfa pancreatiche in cellule beta simili a quelle funzionali.
Genprex (NASDAQ: GNPX) anunció avances en su programa de diabetes a través de una colaboración estratégica con un socio CDMO para investigar un sistema de entrega de nanopartículas lipídicas no virales para su candidato a fármaco de terapia génica para la diabetes. Este enfoque alternativo representa un desarrollo de segunda generación, separado de los programas preclínicos existentes.
El tratamiento actual de la empresa para la diabetes, GPX-002, utiliza un vector AAV que contiene los genes Pdx1 y MafA administrados en el conducto pancreático. Si bien es efectivo, los sistemas de entrega virales no pueden ser readministrados debido a las respuestas inmunitarias. El enfoque no viral podría permitir múltiples tratamientos, optimizando potencialmente los resultados para los pacientes.
Recientes estudios preclínicos mostraron la efectividad de GPX-002 en modelos de ratón NOD de Tipo 1, manteniendo el control de glucosa durante aproximadamente 120 días, significativamente más que la mediana de 17 días observada con los trasplantes de células islotes tradicionales. La terapia se está desarrollando tanto para la diabetes Tipo 1 como Tipo 2, trabajando en transformar las células alfa pancreáticas en células beta funcionales.
Genprex (NASDAQ: GNPX)는 당뇨병 유전자 치료제 후보를 위한 비바이러스성 지질 나노입자 전달 시스템을 연구하기 위해 CDMO 파트너와 전략적 협력을 통해 당뇨병 프로그램의 발전을 발표했습니다. 이 대안적 접근법은 기존의 전임상 프로그램과는 별개의 2세대 개발을 나타냅니다.
회사의 현재 당뇨병 치료제인 GPX-002는 Pdx1 및 MafA 유전자를 포함하는 AAV 벡터를 췌장관에 주입합니다. 효과적이지만, 바이러스 전달 시스템은 면역 반응으로 인해 재투여할 수 없습니다. 비바이러스 접근법은 여러 번의 치료를 가능하게 하여 환자의 결과를 최적화할 수 있습니다.
최근 전임상 연구에서는 GPX-002가 제1형 NOD 마우스 모델에서 약 120일 동안 혈당 조절을 유지하는 효과를 보여주었으며, 이는 전통적인 섬세 세포 이식에서 관찰된 17일의 중간값보다 훨씬 긴 기간입니다. 이 치료법은 제1형 및 제2형 당뇨병 모두에 대해 개발되고 있으며, 췌장 알파 세포를 기능적인 베타 유사 세포로 변환하는 방식으로 작용합니다.
Genprex (NASDAQ: GNPX) a annoncé des avancées dans son programme sur le diabète grâce à une collaboration stratégique avec un partenaire CDMO pour rechercher un système de livraison à nanoparticules lipidiques non virales pour son candidat médicament de thérapie génique contre le diabète. Cette approche alternative représente un développement de deuxième génération, distinct des programmes précliniques existants.
Le traitement actuel de l'entreprise pour le diabète, GPX-002, utilise un vecteur AAV contenant les gènes Pdx1 et MafA administrés dans le canal pancréatique. Bien qu'efficace, les systèmes de livraison viraux ne peuvent pas être réadministrés en raison des réponses immunitaires. L'approche non virale pourrait permettre des traitements multiples, optimisant potentiellement les résultats pour les patients.
Des études précliniques récentes ont montré l'efficacité de GPX-002 dans des modèles murins NOD de type 1, maintenant le contrôle de la glycémie pendant environ 120 jours, ce qui est significativement plus long que la médiane de 17 jours observée avec les transplantations de cellules îlots traditionnelles. La thérapie est en cours de développement pour le diabète de type 1 et de type 2, en transformant les cellules alpha pancréatiques en cellules bêta fonctionnelles.
Genprex (NASDAQ: GNPX) gab bekannt, dass es Fortschritte in seinem Diabetes-Programm durch eine strategische Zusammenarbeit mit einem CDMO-Partner erzielt hat, um ein nicht-virales Lipid-Nanopartikel-Transportsystem für seinen Kandidaten zur Gentherapie gegen Diabetes zu erforschen. Dieser alternative Ansatz stellt eine Entwicklung der zweiten Generation dar, die von bestehenden präklinischen Programmen getrennt ist.
Die aktuelle Diabetesbehandlung des Unternehmens, GPX-002, verwendet einen AAV-Vektor, der die Gene Pdx1 und MafA enthält, die in den Pankreasgang verabreicht werden. Obwohl effektiv, können virale Transportsysteme aufgrund immunologischer Reaktionen nicht erneut verabreicht werden. Der nicht-virale Ansatz könnte mehrere Behandlungen ermöglichen und damit potenziell die Ergebnisse für die Patienten optimieren.
Aktuelle präklinische Studien zeigten die Wirksamkeit von GPX-002 in Typ-1-NOD-Mausmodellen, wobei der Blutzuckerkontrollzeitraum von etwa 120 Tagen aufrechterhalten wurde, was deutlich länger ist als die mediane Dauer von 17 Tagen, die bei traditionellen Inselzelltransplantationen beobachtet wurde. Die Therapie wird sowohl für Typ-1- als auch für Typ-2-Diabetes entwickelt und wirkt, indem sie die Pankreas-Alphazellen in funktionelle beta-ähnliche Zellen umwandelt.
- Preclinical studies show extended glucose control duration of 120 days, significantly exceeding traditional treatments
- Development of non-viral delivery system could enable multiple treatment administrations
- Treatment potentially effective for both Type 1 and Type 2 diabetes
- Current viral delivery system cannot be readministered due to immune response limitations
- Research is still in early exploratory phase
Insights
The announcement of Genprex's research into non-viral delivery systems for their diabetes gene therapy represents a potentially significant technological advancement in the field. The current standard AAV-based delivery systems, while effective, have a critical limitation: they can only be administered once due to immune response development. The exploration of lipid nanoparticle delivery systems could be a game-changer, particularly given their potential for multiple administrations.
The preclinical data showing 120-day glucose control in NOD mice is particularly impressive when compared to the typical 17-day median control achieved with traditional syngeneic islet cell transplants. This 7-fold improvement in durability suggests that Genprex's approach might be creating insulin-producing cells that are sufficiently different from native beta cells to evade autoimmune destruction.
The dual applicability of GPX-002 for both Type 1 and Type 2 diabetes represents a significant market opportunity. The delivery method, requiring only a routine endoscopy procedure, could provide a practical advantage for clinical implementation. However, several critical challenges remain:
- The successful translation of mouse model results to human patients
- The optimization of the lipid nanoparticle formulation for effective gene delivery
- The demonstration of comparable efficacy to the AAV-based approach
- The potential cost implications of a re-dosable therapy
While this research is still in early stages, it positions Genprex at the forefront of innovation in diabetes gene therapy. The company's forward-thinking approach in exploring alternative delivery systems could potentially address key limitations of current gene therapy approaches while opening new possibilities for treatment optimization.
Researching Novel Diabetes Gene Therapy Using Potentially Re-Dosable Non-Viral Delivery System
Strategic Collaboration with CDMO Partner Positions Genprex as a Thought-Leader in Diabetes Market
"This collaboration with our CDMO partner is separate from our existing preclinical research programs and seeks to evaluate potential next generation construct optimization," said Ryan Confer, President and Chief Executive Officer at Genprex. "This exploratory research represents our forward-thinking approach within our diabetes program to determine if our novel diabetes gene therapy could be delivered using a non-viral delivery system, similar to Genprex's oncology drug candidate. While AAV delivery is a well understood delivery mechanism, there are many benefits to a non-viral delivery system, including the potential for re-dosing patients to optimize treatment. This collaboration positions Genprex as a thought-leader in gene therapy, specifically within the diabetes market where there are only symptom managing therapies but no disease modifying technologies."
Separate from the ongoing preclinical studies, Genprex and its CDMO partner are researching and exploring the potential delivery of these genes using a non-viral lipid nanoparticle. Many gene therapies rely on viral based delivery systems. The benefit of the viral system is that viruses are skilled at penetrating cells. However, viruses cannot be readministered, due to the development of an immune response against the virus capsid.
"Prior studies have shown that Non-Obese Diabetic (NOD) mice became hyperglycemic again a median of 17 days after treatment with syngeneic islet cell transplants," said Mark Berger, Chief Medical Officer at Genprex. "Our recent preclinical studies of Type 1 NOD mouse models treated with GPX-002 have shown durability of glucose control to approximately 120 days, or 4 months. This suggests that that the new insulin producing beta-like cells that our therapy produced are poorly recognized by the autoimmune process in NOD mice. Our team continues to explore ways to enhance durability, and expanding our research to include a non-viral delivery system could allow for multiple treatments to be administered."
GPX-002 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.
About Diabetes
According to the
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, 2023.
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 diabetes clinical development program; 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, including the results of our early research to extend durability of glucose control and the potential results of further research with a CDMO to utilize a non-viral delivery system that would allow multiple treatments to be administered; 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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FAQ
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