Policy & Regulation
Genprex and University of Pittsburgh Sign Exclusive License Agreement for Potentially Curative Gene Therapy Candidate for Diabetes
13 February 2020 - - US-based clinical-stage gene therapy company Genprex, Inc. (NASDAQ: GNPX) has signed an exclusive license agreement with the University of Pittsburgh for a diabetes gene therapy that may have the potential to cure Type 1 and Type 2 diabetes, which together currently affect approximately 30.3m people in the U.S, or 9% of the US population, the company said.

The diabetes gene therapy, which was developed by lead researcher and Harvard graduate, Dr. George Gittes, at the Rangos Research Center at UPMC Children's Hospital of Pittsburgh, works by reprogramming beta cells in the pancreas to restore their function, thereby replenishing levels of insulin.

The novel infusion process uses an endoscope and an adeno-associated virus vector to deliver Pdx1 and MafA genes to the pancreas.

The proteins these genes express 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.

The diabetes gene therapy has been tested in vivo in mice and nonhuman primates. In studies of diabetic mice, the gene therapy approach restored normal blood glucose levels for an extended period of time, typically around four months.

According to Dr. Gittes, the duration of restored blood glucose levels in mice could translate to decades in humans.

Following preclinical studies, Dr. Gittes and his team plan to begin a Phase I clinical trial in diabetic patients, which could be the first-ever gene therapy tested in humans for diabetes.

Genprex will add this exciting technology to its research and development pipeline, diversifying its portfolio and expanding its clinical development programs.

The company will continue its focus on developing its immunogene therapies for cancer, including Oncoprex immunogene therapy, its lead drug candidate for non-small cell lung cancer, in parallel with development of the new diabetes gene therapy.

Genprex plans to pursue potential partnerships for the development of this therapy globally and in the US.

According to the American Diabetes Association, more than 30 m Americans have diabetes, and approximately 1.5m Americans are diagnosed with diabetes every year.

Diabetes patients have the continuous burden of checking and monitoring their blood glucose levels and injecting insulin on a daily basis.

Without effective management of diabetes, patients are at risk of stroke, hyperglycemia, cardiovascular disease, diabetic ketoacidosis and extremity amputation. Diabetes is the seventh leading cause of death in the US.

Genprex is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and other serious diseases.

The company's technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and other serious diseases who currently have limited treatment options.

Genprex works with world-class institutions and collaborators to in-license and develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches for patients with cancer and other serious diseases.

The company's lead product candidate, Oncoprex immunogene therapy for non-small cell lung cancer (NSCLC), uses the company's unique, proprietary platform which delivers cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities.

In January 2020, the FDA granted Fast Track Designation for Oncoprex in combination with AstraZeneca's Tagrisso for the treatment of NSCLC.