Type 1 diabetes is a chronic autoimmune disease affecting millions of people globally, characterized by the destruction of insulin-producing beta cells in the pancreas. Patients with Type 1 diabetes currently rely on insulin injections and lifestyle adjustments to manage their blood sugar levels, but these treatments do not address the underlying cause of the disease.
Genprex, a clinical-stage gene therapy company, has recently released groundbreaking data from a non-human primate study evaluating a novel gene therapy to treat Type 1 diabetes. This therapy is part of Genprex's mission to develop life-changing therapies for patients with cancer and diabetes who currently have limited treatment options.
The University of Pittsburgh recently presented data on the potential of Genprex's gene therapy for Type 1 diabetes at the 16th International Conference on Advanced Technologies & Treatments for Diabetes held in Berlin, Germany. During the conference, Ranjeet S. Kalsi, DO, a member of the laboratory of George Gittes, MD, from The University of Pittsburgh's Division of Pediatric Surgery, presented the research findings.
The presentation titled "Pancreatic Intraductal Infusion of Adeno-Associated Virus (AAV) to Treat Non-Human Primates in a Toxin-Induced Diabetes Model" detailed the results from eight non-human primates (NHPs) that had toxin-induced diabetes after receiving streptozocin. The research team administered a novel infusion process that utilized an AAV vector to directly deliver Pdx1 and MafA genes to the pancreas.
According to the study results, after the AAV-engineered construct infusion, eight NHPs had statistically significant decreases in insulin requirements (p<0.001), increases in c-peptide levels (p<0.05), and improvements in glucose tolerance compared to baseline (p<0.05). One of the NHPs demonstrated a normal glucose tolerance test three months after the infusion. Immunohistochemistry also revealed insulin staining, indicating the formation of insulin-producing cells.
The Chief Medical Officer of Genprex, Dr. Mark Berger, said that the recently announced study results are very important because they indicate that the gene therapy generates new insulin-producing beta-like cells in this model of Type 1 diabetes in NHPs. He also noted that the new results confirm previous studies in diabetic mice models, which have already shown positive outcomes. Dr. Berger expressed his excitement about the possibility of conducting human clinical trials to further prove the therapy's effectiveness and to provide long-term beta-cell replacement for patients.
The diabetes technologies used in the study were licensed from the University of Pittsburgh and developed in the laboratory of Dr. George Gittes. Dr. Gittes noted that the statistically significant outcomes from the study support the potential of the gene therapy approach to treating diabetes.
Rodney Varner, the CEO of Genprex, believes that the promising data from the study further supports the potential of the gene therapy approach to change the course of diabetes in both Type 1 and Type 2 diabetes. Based on preclinical in vivo studies, the company is optimistic that its novel gene therapy approach could provide long-term efficacy in humans and potentially make exogenous insulin unnecessary, which would be a significant advancement for the millions of people living with diabetes worldwide.
In addition to its diabetes gene therapy program, Genprex is also advancing novel gene therapies for cancer treatment. The company's lead product candidate, REQORSA (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC). REQORSA has received two FDA Fast Track Designations, and clinical trials have shown promising results.
Genprex's non-viral gene therapy platform and proprietary ONCOPREX Nanoparticle Delivery System are at the forefront of gene therapy research and development. The company works with world-class institutions and collaborators to develop drug candidates and provide novel treatment approaches for patients with cancer and diabetes.
With the promising results from the non-human primate study, Genprex's diabetes gene therapy program is a key focus for the company's future growth and success. The potential to provide a new and effective treatment option for patients with Type 1 diabetes is a significant step forward in the fight against this debilitating disease. To learn more about Genprex, visit their website at Genprex.com
