Genprex Inc revealed that it has struck an exclusive license agreement with the University of Pittsburgh for a worldwide, exclusive license to some patent applications and a worldwide, non-exclusive license to use certain related know-how, all related to modulating autoimmunity in Type 1 diabetes by using gene therapy.
The Austin, Texas-based clinical-stage gene therapy company focused on developing novel therapies for patients with cancer and diabetes, noted that the preclinical technology transforms macrophages enabling them to reduce autoimmune activity in Type 1 diabetes and could be complementary to the company's existing diabetes technology.
"Gaining exclusive access to technology that modulates the immune system by transforming macrophages could prove to be significant to our broader research partnership with the laboratory of George Gittes, Professor of Surgery and Pediatrics and Chief of the Division of Pediatric Surgery at the University of Pittsburgh School of Medicine," Genprex Chief Medical Officer Mark Berger said in a statement.
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"We are making significant strides in our program with Dr Gittes's innovative approach to treating diabetes by the transformation of alpha cells into beta-like cells and are excited to add to our arsenal this additional technology also out of Dr Gittes's lab, in collaboration with the laboratory of Dr Xangwei Xiao, Assistant Professor of Surgery, also in the Division of Pediatric Surgery at the University of Pittsburgh's School of Medicine,” he added.
Berger noted that the new approach could not only be used to reduce autoimmune activity in Type 1 diabetes by modulating the immune system, but it could potentially work in conjunction with the technology Genprex has licensed previously.
Gene therapy is compelling
"With diabetes reaching epidemic proportions around the world, the work Dr Gittes is pursuing in diabetes is absolutely critical. In the US alone, there are more than 37 million people with diabetes (approximately 1.9 million of whom have Type 1 diabetes) and another approximately 96 million Americans who are pre-diabetic or have abnormally elevated blood sugar levels,” said Genprex CEO Rodney Varner.
“The opportunity to change the course of this disease with gene therapy is extremely compelling, and increasing our exclusive access to intellectual property could prove to be pivotal to our pathway forward," he added.
Genprex signed an exclusive license agreement with the University of Pittsburgh in 2020. The gene therapy approach under the original license is comprised of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes directly to the pancreas.
According to the company, in models of Type 1 diabetes, these genes express proteins that 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. In Type 2 diabetes, where autoimmunity is not at play, it is believed that exhausted beta cells will be rejuvenated and replenished.
This gene therapy approach was developed by Dr Gittes. His preclinical research in this area has been published in scientific publications, and he has won several research grants, including a $2.59 million grant from the National Institutes of Health (NIH) National Institute of Diabetes and Digestive and Kidney Diseases.
Earlier studies in diabetic mouse models showed that gene therapy restored normal blood glucose levels for an extended period of time, typically around four months. It is believed that the duration of restored blood glucose levels in mice could translate to decades in humans. Preliminary data from a more recent study in a non-human primate model of Type 1 diabetes have also been promising. Data from this study are expected to be presented at a scientific meeting during the first quarter of 2023, said the company.
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.
Contact the author Uttara Choudhury at uttara@proactiveinvestors.com
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