Core Viewpoint - Genprex, Inc. has achieved significant clinical development milestones in 2025, focusing on advancing its gene therapy programs for cancer and diabetes, with ongoing clinical trials for lung cancer treatments [1][2]. Group 1: Company Achievements - The company has issued a stockholder letter summarizing recent achievements and outlining key milestones for 2025 and beyond [1]. - Genprex's lead product candidate, Reqorsa® Gene Therapy, is currently being evaluated in two clinical trials for non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC), both of which have received Fast Track Designation from the FDA [3]. - The SCLC program has also received Orphan Drug Designation from the FDA, indicating its potential to address unmet medical needs [3]. Group 2: Technology and Approach - Genprex utilizes a systemic, non-viral Oncoprex® Delivery System that encapsulates gene-expressing plasmids in lipid-based nanoparticles, administered intravenously to target tumor cells [3]. - The diabetes gene therapy approach involves a novel infusion process using an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas, aiming to transform alpha cells into functional beta-like cells capable of producing insulin [3]. Group 3: Future Outlook - The company is focused on executing upcoming milestones in the second half of 2025 and beyond, with a commitment to transforming the lives of patients battling cancer and diabetes through innovative gene therapies [2].

Core Viewpoint - Genprex, Inc. has presented promising preclinical research on its diabetes gene therapy candidate GPX-002, demonstrating its potential to improve glucose homeostasis by reprogramming alpha cells into beta-like cells, as showcased at the 2025 American Diabetes Association Scientific Session [1][2]. Group 1: Research Findings - The research indicates that alpha cells in animal models of Type 1 diabetes (T1D) can transdifferentiate into beta-like cells after being treated with GPX-002, maintaining improved glucose control for three months [2][6]. - The gene therapy utilizes recombinant adeno-associated virus (rAAV) to deliver Pdx1 and MafA genes directly into the pancreatic duct, effectively converting alpha cells into insulin-secreting beta-like cells without the need for immunosuppression in mouse models [3][12]. - In non-human primate studies, the infusion of rAAV resulted in improved glucose tolerance and reduced insulin requirements one month post-infusion, with ongoing evaluations of immune responses to the therapy [5][8]. Group 2: Clinical Development - Genprex is advancing GPX-002 for both Type 1 and Type 2 diabetes, with the same gene therapy approach applied to both conditions, aiming to rejuvenate exhausted beta cells in Type 2 diabetes [9][12]. - The company is currently conducting preclinical studies to gather additional data on the efficacy of GPX-002 after six months of immunosuppression [8][9]. - The therapy is designed to be administered via a routine endoscopy procedure in humans, enhancing its potential for clinical application [9]. Group 3: Company Overview - Genprex, Inc. is a clinical-stage gene therapy company focused on developing innovative therapies for cancer and diabetes, collaborating with leading institutions to advance its drug candidates [10][11]. - The company’s oncology program includes the Oncoprex® Delivery System, which encapsulates gene-expressing plasmids for intravenous administration, targeting tumor cells [11]. - Genprex aims to provide new treatment options for patients with limited alternatives, leveraging its gene therapy technologies [10].

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 ...

Company Overview - Genprex, Inc. is a clinical-stage gene therapy company focused on developing therapies for cancer and diabetes [4] - The company utilizes a systemic, non-viral Oncoprex® Delivery System to administer gene therapies [4] - Genprex's lead product candidate, Reqorsa® Gene Therapy, is being evaluated in clinical trials for non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC) [4] - The company has received Fast Track Designation from the FDA for its lung cancer programs and Orphan Drug Designation for its SCLC program [4] Upcoming Events - Genprex will participate in the BIO 2025 International Convention from June 16-19, 2025, in Boston, Massachusetts [1][3] - Thomas Gallagher, Senior Vice President of Intellectual Property and Licensing, will be available for one-on-one meetings during the conference [2] Technology and Innovation - Genprex's diabetes gene therapy approach involves a novel infusion process using an AAV vector to deliver Pdx1 and MafA genes to the pancreas [4] - In Type 1 diabetes models, GPX-002 transforms alpha cells into functional beta-like cells capable of producing insulin [4] - For Type 2 diabetes, GPX-002 is believed to rejuvenate and replenish exhausted beta cells [4] Industry Context - The BIO International Convention is the largest biotechnology event, attracting 20,000 industry leaders globally [3] - Genprex collaborates with world-class institutions to develop its drug candidates and expand its gene therapy pipeline [4]

Core Viewpoint - Genprex, Inc. is advancing its diabetes gene therapy candidate GPX-002, which shows promise in treating both Type 1 and Type 2 diabetes, as evidenced by positive preclinical data presented at the ASGCT Annual Meeting [1][2]. Group 1: Research and Development - GPX-002 utilizes recombinant adeno-associated virus (rAAV) to deliver Pdx1 and MafA genes, converting alpha cells into beta-like cells that secrete insulin, effectively reversing diabetes in mouse models without the need for immunosuppression [2][3]. - The therapy has been tested in cynomolgus macaques with streptozotocin-induced diabetes, demonstrating improved glucose tolerance and reduced insulin requirements one month post-infusion [4][5]. - Ongoing preclinical studies are evaluating the long-term efficacy of GPX-002 after six months of immunosuppression in non-human primate models for both Type 1 and Type 2 diabetes [6]. Group 2: Presentation Details - The oral presentation at the ASGCT Annual Meeting was titled "Immune Modulation Sustains Alpha Cell Reprogramming and Mitigates Immune Responses to AAV in a Diabetic Non-Human Primate Model," presented by Dr. Hannah Rinehardt [2]. - The presentation highlighted the challenges of managing immune responses to the therapy, indicating that temporary immunosuppression can effectively prevent anti-viral immunity but may need to be extended beyond three months [4][5]. Group 3: Company Overview - Genprex, Inc. is a clinical-stage gene therapy company focused on developing innovative therapies for cancer and diabetes, utilizing advanced technologies to deliver disease-fighting genes [8]. - The company’s approach for diabetes involves a novel infusion process using an AAV vector to directly administer therapeutic genes into the pancreas, with the potential for routine endoscopic procedures in humans [7][8].

Core Viewpoint - Genprex, Inc. has announced a new Sponsored Research Agreement (SRA) with the University of Pittsburgh to further study its gene therapy GPX-002 for Type 1 and Type 2 diabetes in animal models, following the completion of a previous two-year agreement [1][2]. Company Overview - Genprex, Inc. is a clinical-stage gene therapy company focused on developing therapies for cancer and diabetes, utilizing innovative technologies to deliver disease-fighting genes [5][6]. - The company’s lead product candidate, Reqorsa® Gene Therapy, is under evaluation in clinical trials for non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC) [5]. Research and Development - The new SRA with the University of Pittsburgh will sponsor preclinical studies of GPX-002, which has shown promising results in reducing insulin requirements and improving glucose tolerance in animal models of diabetes [2][3]. - GPX-002 employs an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes directly to the pancreas, aiming to transform alpha cells into functional beta-like cells in Type 1 diabetes and rejuvenate exhausted beta cells in Type 2 diabetes [3][6]. Market Context - As of 2024, approximately 38.4 million Americans have diabetes, with 10% having Type 1 diabetes and 90-95% having Type 2 diabetes. The global diabetes population is projected to rise from 537 million in 2021 to 783 million by 2045 [4]. - Diabetes caused over 6.7 million deaths globally in 2021 and resulted in approximately $966 billion in health expenditures, reflecting a significant increase over the past fifteen years [4].

Core Viewpoint - Genprex, Inc. announced positive preclinical data for Reqorsa® Gene Therapy, demonstrating its robust anti-tumor effects in KRASG12C mutant non-small cell lung cancer (NSCLC), both alone and in combination with LUMAKRAS® [1][2]. Group 1: Preclinical Data Presentation - The positive preclinical data was presented at the 2025 American Association for Cancer Research (AACR) Annual Meeting, highlighting the therapeutic potential of REQORSA in treating Ras inhibitor resistant lung cancer [1][2]. - The poster titled "Overcoming sotorasib acquired resistance in KRASG12C mutant NSCLC by TUSC2 gene therapy" showcased that TUSC2 gene therapy effectively overcomes acquired resistance to sotorasib in mouse xenografts [3]. Group 2: Efficacy of Reqorsa - TUSC2 transfection significantly reduced colony formation and increased apoptosis in acquired resistance cell lines, indicating strong anti-tumor efficacy [3]. - REQORSA alone showed a strong anti-tumor effect on TC314AR PDXs, while sotorasib alone exhibited no significant activity [3]. - A synergistic effect was observed when combining REQORSA with sotorasib, enhancing tumor control compared to either treatment alone [3]. Group 3: Technology and Delivery System - Reqorsa consists of a plasmid containing the TUSC2 gene encapsulated in non-viral lipid-based nanoparticles, specifically targeting cancer cells while minimizing uptake by normal tissue [5]. - Laboratory studies indicated that the uptake of TUSC2 in tumor cells after REQORSA treatment was 10 to 33 times higher than in normal cells [5]. Group 4: Company Overview - Genprex, Inc. is a clinical-stage gene therapy company focused on developing therapies for cancer and diabetes, utilizing its Oncoprex® Delivery System for gene administration [6][7]. - The company is advancing its pipeline of gene therapies and has received Fast Track Designation from the FDA for its lung cancer clinical programs [7].

Core Viewpoint - Genprex, Inc. has announced the presentation of positive preclinical data for its diabetes gene therapy candidate GPX-002 at the upcoming ASGCT Annual Meeting, highlighting the potential of gene therapy as a curative treatment for diabetes [1][2]. Group 1: Research and Development - The oral presentation will focus on the immune modulation and reprogramming of alpha cells to beta-like cells in a diabetic non-human primate model, showcasing the effectiveness of the rAAV gene therapy approach [3]. - The study demonstrated that retrograde intraductal infusion of rAAV6 can convert alpha cells into insulin-secreting beta-like cells, reversing diabetes in mouse models [4]. - One month post-infusion, non-human primates exhibited improved glucose tolerance and reduced insulin requirements, indicating the therapy's durable effects [5]. Group 2: Immune Response Management - The research evaluated the immune response to direct infusion of rAAV into the pancreatic duct and how to manage potential anti-viral immunity, which can hinder gene therapy success [4][5]. - Temporary immunosuppression using a combination of rituximab, rapamycin, and steroids was largely effective in preventing anti-viral immune responses, allowing sustained therapeutic effects [6]. Group 3: Product Overview - GPX-002 is being developed for both Type 1 and Type 2 diabetes, utilizing an AAV vector to deliver Pdx1 and MafA genes directly into the pancreatic duct [7]. - In Type 1 diabetes, GPX-002 aims to transform alpha cells into functional beta-like cells, while in Type 2 diabetes, it is believed to rejuvenate exhausted beta cells [7]. Group 4: Company Background - Genprex, Inc. is a clinical-stage gene therapy company focused on developing therapies for cancer and diabetes, collaborating with leading institutions to advance its drug candidates [8].

Core Viewpoint - Genprex, Inc. has signed an exclusive patent license agreement with NYU Langone Health for Reqorsa® Gene Therapy, which is being investigated as a treatment for mesothelioma, thereby expanding its oncology portfolio and clinical development pipeline [1][2]. Company Overview - Genprex, Inc. is a clinical-stage gene therapy company focused on developing therapies for cancer and diabetes, utilizing its Oncoprex® Delivery System to administer disease-fighting genes [8][9]. - The company collaborates with leading research institutions to advance its gene therapy candidates [8]. Product Details - Reqorsa® Gene Therapy (quaratusugene ozeplasmid) is designed to deliver the TUSC2 gene to cancer cells, with studies showing a 10 to 33 times higher uptake in tumor cells compared to normal cells [7][9]. - The therapy has demonstrated significant decreases in cell proliferation and invasion, along with increased apoptosis in malignant pleural mesothelioma cell lines [4]. Research and Development - Positive preclinical data for Reqorsa was presented at the 2024 EORTC-NCI-AACR Symposium, indicating its potential effectiveness against mesothelioma [2][4]. - Genprex has established a Mesothelioma Clinical Advisory Board to support its preclinical oncology program, consisting of four renowned researchers [5]. Market Context - Mesothelioma is a rare cancer with approximately 3,000 new cases diagnosed annually in the U.S., primarily affecting the pleura, which accounts for 80-90% of cases [6]. - The life expectancy for pleural mesothelioma is around 18 months, with a 3-year survival rate of 23% for treated patients [6].

Core Viewpoint - Genprex, Inc. announced positive preclinical data for its lead drug candidate, Reqorsa® Gene Therapy, targeting KRASG12C mutant non-small cell lung cancer (NSCLC), which may provide a new treatment option for patients resistant to Ras inhibitors [1][2]. Group 1: Research and Development - The upcoming presentation at the 2025 American Association for Cancer Research (AACR) Annual Meeting will showcase preclinical data on Reqorsa® Gene Therapy [1]. - The study focuses on overcoming acquired resistance to sotorasib (Lumakras®), the first FDA-approved KRAS inhibitor, in KRASG12C mutant NSCLC [3]. - TUSC2, the active component in Reqorsa, has shown multifunctional activity, including inhibiting cancer cell growth and activating immune responses [3][4]. Group 2: Clinical Efficacy - TUSC2 transfection significantly reduced colony formation and increased apoptosis in acquired resistance (AR) cell lines [4]. - Reqorsa demonstrated a strong antitumor effect in mouse xenografts and patient-derived xenograft (PDX) models, outperforming sotorasib alone [4][5]. - A synergistic effect was observed when combining Reqorsa with sotorasib in treating TC314AR PDX tumors [4]. Group 3: Product Information - Reqorsa (quaratusugene ozeplasmid) is a gene therapy that delivers the TUSC2 gene using a non-viral lipid-based nanoparticle system, specifically targeting cancer cells [6][8]. - Laboratory studies indicate that TUSC2 uptake in tumor cells is significantly higher than in normal cells, ranging from 10 to 33 times [7]. Group 4: Company Overview - Genprex, Inc. is a clinical-stage gene therapy company focused on developing therapies for cancer and diabetes, utilizing its Oncoprex® Delivery System [8]. - The company is advancing its pipeline of gene therapies and has received Fast Track Designation from the FDA for its lung cancer programs [8].