Genprex, Inc. (Genprex), a clinical-stage gene therapy company, announced that following the recent completion of its two-year Sponsored Research Agreement (SRA) with the University of Pittsburgh of the Commonwealth System of Higher Education (Pitt) to study Type 1 diabetes (T1D) and Type 2 diabetes (T2D), the parties have entered into a new SRA to study GPX-002, the company’s gene therapy for diabetes, in T1D and T2D in animal models.
“We continue to make advancements in our studies of GPX-002 in both T1D and T2D, including mouse models, construct optimization and studies in other animal models,” said Ryan Confer, president and chief executive officer at Genprex. “As we advance into new animal studies in both T1D and T2D, we look forward to exploring how GPX-002 may decrease insulin requirements and improve glucose tolerance. We believe these studies set us on a path toward human clinical trials.”
Genprex has entered into a new SRA with Pitt in which the Company will sponsor preclinical studies of GPX-002 for the treatment of T1D and T2D in animal models. Preclinical data from an animal study of GPX-002 in T1D showed statistically significant decreases in insulin requirements, increases in c-peptide levels and improvements in glucose tolerance in the treated animals compared to baseline. These groundbreaking results support the hypothesis that this disruptive gene therapy approach developed by Pitt researchers may be a promising treatment for both T1D and T2D.
“We are excited to continue our pioneering gene therapy work in diabetes through this SRA,” said Mark Berger, MD, chief medical officer of Genprex. “Our preclinical studies have produced compelling data that have encouraged us to support the expansion of this promising gene therapy approach to treat T2D with GPX-002 in animal models.”
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.
According to the US Center for Disease Control and Prevention as of 2024, 38.4 million Americans, or approximately 11.6% of the US population, have diabetes. Approximately 10% of this total has T1D and approximately 90-95% of this total has T2D. It is also believed that more than 97 million Americans aged 18 years or older have prediabetes. In 2021, approximately 537 million adults (20-79 years) worldwide were living with diabetes, and the total number of people living with diabetes is projected to rise to 643 million by 2030 and 783 million in 2045. Approximately 7.7 million adults over the age of 20 live with T1D worldwide, and approximately 1.6 million children and adolescents under the age of 20 live with T1D worldwide. Also in 2021, diabetes caused more than 6.7 million deaths globally and diabetes resulted in approximately $966 billion dollars in health expenditures, a 316% increase over the preceding fifteen years.
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.