Streptozocin

AUSTIN, TX / ACCESSWIRE / April 19, 2023 / On their own, cancer or diabetes can be debilitating and deadly. The two are common diseases with tremendous impact on health worldwide. To make matters worse, epidemiologic evidence suggests that people with diabetes - especially Type 2 diabetes - are at significantly higher risk for many forms of cancer. The two pose a significant health problem and threat globally. In oncology, Genprex, Inc.(NASDAQ: GNPX) is initially focused on lung cancer. Annually, there are more than 2 million new cases of lung cancer worldwide with an average five-year survival rate of 18.6%. Over 537 million people worldwide suffer from diabetes with annual global mortality of about 6.7 million. Gene Therapy To The Rescue? Genprex Inc., a clinical-stage gene therapy company, is focused on developing for these patients with serious medical conditions and unmet need new and better treatment options that are life-changing. Founded in 2009 and headquartered in Austin, Texas, the company has been at the forefront of gene therapy research, focusing on harnessing the power of cutting-edge technologies to improve the lives of those affected by debilitating conditions. Driven by a deep commitment to scientific excellence and patient-centricity, Genprex reports that its mission is to revolutionize the treatment landscape by developing innovative gene therapies that have the potential to transform the lives of patients who have limited treatment options. Innovative Pipeline Genprex's oncology program utilizes the company's proprietary, non-viral ONCOPREX® Nanoparticle Delivery System, which it believes is the first systemic gene therapy delivery platform used for cancer in humans. ONCOPREX® encapsulates the gene-expressing plasmids using lipid nanoparticles, and the resultant product is administered intravenously, where it is then taken up by tumor cells in which tumor suppressor genes are missing or deficient. Genprex's value proposition lies in its robust and diversified pipeline of gene therapy candidates addressing large underserved patient populations, a strong intellectual property portfolio, and a seasoned management team with extensive experience in biopharmaceutical development and commercialization. The company's pipeline is anchored by its lead product candidate, REQORSA™ Immunogene Therapy (quaratusugene ozeplasmid), an innovative gene therapy that has been shown in pre-clinical testing to be potentially applicable to many types of cancer. Genprex is initially targeting non-small cell lung cancer (NSCLC) patients and small cell lung cancer (SCLC). REQORSA™ works by introducing a tumor suppressor gene (TUSC2) into cancer cells, inhibiting tumor growth and promoting cancer cell death. REQORSA™is being evaluated by Genprex as a treatment for NSCLC in two phase 1/2 clinical trials, with each trial receiving a Fast Track Designation ("FTD") from the U.S. Food and Drug Administration (FDA). One FTD is for use of REQORSA in combination with Merck & Co's Keytruda®, and the other is for use of REQORSA in combination with Astra Zeneca's Tagrisso REQORSA is also being evaluated for SCLC in combination with Genentech's Tecentriq with a trial expected to launch later this year. REQORSA has shown promise in preclinical and clinical studies, demonstrating its potential to improve patient outcomes in combination with other cancer treatments. Genprex sees the potential to use its proprietary gene therapy delivery platform to pursue a number of other cancer indications targeting the delivery of therapeutic genes to tumors as opposed to normal cells. The company's diabetes gene therapy approach comprises a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. In models of Type 1 diabetes, the genes express proteins that transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body's immune system. In Type 2 diabetes, where autoimmunity is not at play, it is believed that exhausted beta cells are rejuvenated and replenished. The company in February announced groundbreaking data from a non-human primate study evaluating its novel gene therapy to treat Type 1 diabetes. Results showed statistically significant decreases in insulin requirements, increases in c-peptide levels and improvements in glucose tolerance compared to baseline. The report was based on results from eight non-human primates (NHPs) with toxin-induced diabetes after streptozocin administration. They received a novel infusion process that used an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas. Big Pharma's Bet On Gene Therapy Big Pharma is rapidly accelerating investment in emerging gene therapies with diabetes being of particular note. Major players, such as Eli Lilly and Company, Sanofi SA, and Novo Nordisk A/S, are advancing novel therapies that may supplant traditional insulin-based therapies. In April 2021, Jaguar Gene Therapy announced the closing of a $139 million Series B investment led by Eli Lilly and Company and Deerfield Management, to support the advancement of a novel gene therapy for type 1 diabetes. In March 2023, Sanofi announced the intention to acquire Provention Bio, and its TZIELD therapy that delays the onset of Type 1 diabetes, in a deal valued at approximately $2.9 billion. In April 2023, Novo Nordisk entered a collaboration with Aspect Biosystems to pursue novel therapeutics for Type 1 diabetes and obesity, in a deal valued at approximately $2.6 billion. Other big players like Amgen Inc., Gilead Sciences Inc., Biogen Inc., and bluebird bio Inc., have also made big bets in gene therapy. Intellectual Property Genprex's intellectual property portfolio - consisting of numerous issued patents and pending patent applications licensed from major academic research institutions - could provide the company with a strong competitive advantage in the gene therapy space. This IP protection covers key aspects of the company's proprietary technologies and product candidates, including methods of gene delivery, therapeutic compositions and manufacturing processes. Experienced Management Team The success of Genprex is driven by its experienced and dedicated management team, which brings decades of combined experience in biopharmaceutical development, commercialization, and business operations. This expertise could ensure that the company is well-positioned to navigate the complex landscape of gene therapy development and deliver on its mission to bring innovative treatments to large populations of patients in need. Genprex Strengthens Diabetes Gene Therapy Program On December 15 last year, the company announced it had entered into an exclusive license agreement with the University of Pittsburgh, granting Genprex a worldwide, exclusive license to certain patent applications and related technology and a worldwide, non-exclusive license to use certain related know-how, all related to modulating autoimmunity in Type 1 diabetes by using gene therapy. The preclinical technology transforms macrophages, enabling them to reduce autoimmune activity in Type 1 diabetes, and could be complementary to the company's existing diabetes technology. Genprex Signs Exclusive License To Additional Diabetes Technology The company also revealed on January 5 that it has entered into another exclusive license agreement with the university, granting Genprex a worldwide, exclusive license to a patent application and related technology and a worldwide, non-exclusive license to use certain related know-how, all related to gene therapy for Type 2 diabetes using the genes of the Pdx1 and MafA transcription factors. The preclinical technology, GPX-003, is believed to work by rejuvenating diminished beta cells to increase insulin expression by introducing transcription factors controlled by an insulin promoter. Featured photo sourced from Shutterstock CONTACT: Genprex investors@genprex.com SOURCE: Genprex View source version on accesswire.com:

A female Researcher works with vials of blood/courtesy of Getty Images Genome editing has been a hot-button topic in recent years. With the rise of CRISPR technology in the news, this is not surprising for either scientists or investors. In February 2022, CRISPR Therapeutics and ViaCyte made history, carrying out the first-in-human transplant of gene-edited, stem cell-derived pancreatic cells to treat Type 1 diabetes (T1D). With results of an animal study published in February 2023, Genprex aims to add credibility to another genome editing approach in the hunt for a functional cure to T1D. Based in Austin, Texas, Genprex focuses on developing life-changing therapies for cancer and diabetes. The company’s pipeline includes three different products under investigation: Reqorsa, GPX-002 and GPX-003. While Reqorsa is seeking indications in cancer in Phase I trials, GPX-002 and GPX-003 are undergoing preclinical trials for diabetes, with GPX-002 targeting T1D and GPX-003 targeting type 2 diabetes (T2D). While traditional gene therapies include replacement gene therapy, gene editing and CAR-T for diabetes, Genprex aims to use Adeno-Associated Virus (AAV) pancreatic intraductal infusions to deliver Pdx1 and MafA genes to the pancreas. Non-Human Primate Study One month after eight non-human primates (NHPs) were injected with streptozocin to induce diabetes, surgical procedures were performed to provide infusions to the subjects. Results showed the NHPs had decreased insulin requirements, increased C-peptide levels, improved glucose tolerance compared to baseline and more insulin-positive cells compared to non-treated diabetic controls based on immunohistochemistry. The researchers assessed blood glucose and insulin requirements. During the untreated diabetes induction period, total daily exogenous insulin levels were between 8 and 10 units and average daily blood glucose levels were between 50 and 100 mg/dL. After Genprex’s investigational AAV-CMV-Pdx1-mafA construct was injected during the surgical procedure, average daily blood glucose levels spiked to 200 mg/dL initially, but total daily exogenous insulin levels decreased to below 4 units. Average daily blood glucose levels decreased to around 100 mg/dL 56 days after the operation. Average glucose stimulated insulin secretion remained higher following the operation compared to prior, and eight weeks after the operation, glucose suppression capabilities improved. At the 3-month mark, NHP2 recent glucose tolerance test results showed there was virtually no difference between treated diabetic NHPs at the 3-month mark versus healthy NHPs. While this achievement may seem to be revolutionary, there are unknowns that need to be assessed. Genprex stated the gene therapy could be administered without an operation using routine endoscopic retrograde cholangiopancreatography in humans, but this is a different type of procedure from the one used on NHPs. In addition, the allure of a quick cure to diabetes may require some cultural acceptance for patients used to lifelong interventions such as oral and injectable treatments. Finally, Genprex’s human trials have yet to commence, so FDA approval for this type of treatment may not hit the market until the latter half of the 2020s at the earliest. CRISPR and ViaCyte in Phase I CRISPR and ViaCyte are slightly further ahead with their diabetes genome editing treatment, VCTX210. This was a key attraction for Vertex, which acquired ViaCyte in July 2022. While previous products from ViaCyte such as PEC-Direct require long-term immunosuppression with drugs, VCTX210 is designed to be a more permanent solution without the need for immunosuppressants. As of February 2023, the companies had completed dosing in Phase I subjects. While this is technically one step ahead of Genprex, the Phase I trial is primarily looking at safety as opposed to efficacy data. There are also some differences between VCTX210 and Genprex’s products. VCTX210 is an allogeneic, gene-edited, stem cell-derived therapy designed as a best-in-class treatment for T1D and insulin-dependent T2D, while Genprex uses viral delivery. Regardless of what the future holds for Genprex versus CRISPR/Viacyte, these gene therapy agents hold much promise in the search for a single solution to diabetes. With potential answers for a patient pool that numbers in the hundreds of millions worldwide, keep an eye out for these frontrunners in the next few years.