Genprex, Inc., a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, announced the advancement of its diabetes program through a strategic collaboration with a contract development and manufacturing organization (CDMO) to research an alternative second generation approach using a non-viral lipid nanoparticle delivery of the Company's diabetes gene therapy drug candidate.

"This collaboration with our CDMO partner is separate from our existing preclinical research programs and seeks to evaluate potential next generation construct optimization," said Ryan Confer, President and Chief Executive Officer at Genprex. "This exploratory research represents our forward-thinking approach within our diabetes program to determine if our novel diabetes gene therapy could be delivered using a non-viral delivery system, similar to Genprex's oncology drug candidate. While AAV delivery is a well understood delivery mechanism, there are many benefits to a non-viral delivery system, including the potential for re-dosing patients to optimize treatment. This collaboration positions Genprex as a thought-leader in gene therapy, specifically within the diabetes market where there are only symptom managing therapies but no disease modifying technologies."

Separate from the ongoing preclinical studies, Genprex and its CDMO partner are researching and exploring the potential delivery of these genes using a non-viral lipid nanoparticle. Many gene therapies rely on viral based delivery systems. The benefit of the viral system is that viruses are skilled at penetrating cells. However, viruses cannot be readministered, due to the development of an immune response against the virus capsid.

"Prior studies have shown that Non-Obese Diabetic (NOD) mice became hyperglycemic again a median of 17 days after treatment with syngeneic islet cell transplants," said Mark Berger, Chief Medical Officer at Genprex. "Our recent preclinical studies of Type 1 NOD mouse models treated with GPX-002 have shown durability of glucose control to approximately 120 days, or 4 months. This suggests that that the new insulin producing beta-like cells that our therapy produced are poorly recognized by the autoimmune process in NOD mice. Our team continues to explore ways to enhance durability, and expanding our research to include a non-viral delivery system could allow for multiple treatments to be administered." 

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.