Genprex Announces Groundbreaking Data from Non-Human Primate Study Evaluating Novel Gene Therapy to Treat Type 1 Diabetes

Genprex, a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that data highlighting the potential of Genprex’s gene therapy for Type 1 diabetes is being presented by its research collaborators at the University of Pittsburgh at the 16th International Conference on Advanced Technologies & Treatments for Diabetes (ATTD 2023) taking place February 22-25, 2023 in Berlin, Germany.

The abstract data were released, and the presentation will be delivered on February 25th at 2:25 pm CET by Ranjeet S. Kalsi, DO, a member of the laboratory of George Gittes, MD, from Pitt’s Division of Pediatric Surgery, Pittsburgh, Pennsylvania. The complete presentation will be available on the Company’s website here following the close of Dr. Kalsi’s presentation. The presentation titled, “Pancreatic Intraductal Infusion of Adeno-Associated Virus (AAV) to Treat Non-Human Primates in a Toxin-Induced Diabetes Model,” will report 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.

“These results are compelling as they demonstrate the potential for this gene therapy to create newly formed beta-like cells that can produce insulin. They also validate earlier studies of this approach in diabetic mouse models that showed restoration of normal blood glucose levels for several months,” stated Mark Berger, MD, Chief Medical Officer of Genprex. “We are eager to continue working to advance this gene therapy into human clinical trials in order to replicate these outcomes in people and potentially provide long-term replacement of beta-cells.”

The statistically significant study results show that post-infusion of the AAV engineered construct, the eight NHPs had “decreased insulin requirements (p<0.001); increased c-peptide levels (p<0.05); and improved glucose tolerance compared to baseline (p<0.05) with one demonstrating reestablished normoglycemia. Immunohistochemistry revealed insulin and glucagon staining, which suggest the formation of insulin-producing cells.”

The diabetes technologies licensed from Pitt by Genprex were developed in the laboratory of George Gittes, MD, Professor of Surgery and Pediatrics and Chief of the Division of Pediatric Surgery at the University of Pittsburgh School of Medicine. “We are delighted with the statistically significant outcomes from this NHP study in Type-1 diabetes as it further supports our thesis on the potential of our gene therapy approach in diabetes. Importantly, it shows a positive data progression from our earlier mouse model data to these NHP data,” noted Dr. Gittes.

“These promising data are very encouraging and further support our belief that this gene therapy approach to treating diabetes has the potential to change the course of the disease in both Type 1 and Type 2 diabetes,” said Rodney Varner, President and Chief Executive Officer of Genprex. “Based on data from a number of preclinical in vivo studies, we are encouraged that our novel gene therapy approach could provide long-term efficacy in humans potentially making exogenous insulin unnecessary, which would be a gamechanger for the 537 million people around the world living with diabetes.”