Latest Research Demonstrates Promising Improved Glucose Homeostasis by Reprogramming Alpha Cells
AUSTIN, TX, USA I June 24, 2025 I Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that its research collaborators presented positive preclinical research from studies of GPX-002, the Company’s diabetes gene therapy drug candidate, at the 2025 American Diabetes Association (ADA) 85th Scientific Session in Chicago.
“We are proud of the research presented at the ADA conference, which demonstrates that alpha cells in animal models of T1D have undergone transdifferentiation to beta-like cells after being transduced with GPX-002. In addition, the beta-like cells were still providing improved control of glucose levels after three months,” said Ryan Confer, President and Chief Executive Officer at Genprex. “We are very excited to continue evaluating GPX-002 as we further optimize how to control anti-viral immunity and move toward human clinical trials.”
The oral presentation details for the Genprex-supported abstract at the ADA 85th Scientific Sessions:
Title: Recombinant AAV-mediated Gene Therapy For The Treatment Of Streptozotocin-Induced Diabetes in Non-Human Primates
Type: Oral Presentation
Track: Immunology/Beta-Cell Replacement
Category: Exploring Alternative Strategies to Stem Cell-Derived Beta Cells and Insights from Autologous Islet Transplantation
Presentation Date: Monday, June 23, 2025
Presentation Time: 1:30 p.m. CT
Presenter: Hannah Rinehardt, MD, University of Pittsburgh Medical Center
The oral presentation discussed GPX-002, the diabetes gene therapy, which uses infusion of recombinant adeno-associated virus (rAAV) retrograde into the pancreatic duct to deliver the Pdx1 and MafA genes. The gene therapy converts alpha cells into beta-like cells that secrete insulin physiologically, reversing diabetes in mouse models, where immunosuppression was not necessary. Researchers evaluated the immune response to direct infusion of rAAV into the pancreatic duct of non-human primates (NHPs) with streptozotocin-induced diabetes and evaluated how to best manage immune responses against the virus capsid proteins.
Diabetes was induced with streptozotocin (STZ) in cynomolgus macaques, a type of NHP. NHPs received retrograde intraductal infusion of rAAV via laparotomy for precise delivery to the pancreas. rAAV capsids were chosen based on tropism for endocrine cells, and pre-existing neutralizing antibody titers (NAbs) were negative.
Expression of viral proteins occurs for a limited period of time after rAAV infection, since the infection doesn’t produce new AAV virus. One-month post-infusion, NHPs showed improved glucose tolerance and reduced insulin requirements. In the following months, using steroid-sparing regimens, increases in pancreatic B and T lymphocyte populations were noted on single cell RNA sequencing. Temporary immunosuppression (IS), using a combination of rituximab, rapamycin, and steroids for a 3-month course is largely effective at preventing anti-viral immunity. However, discontinuation of IS at 3 months post-infusion led to an immune response afterwards, indicating that IS in NHPs may need to be continued longer, and six months of IS in NHPs is now being evaluated.
When colocalization of insulin and glucagon is quantified, there was significantly elevated colocalization in treated islets compared to untreated diabetic and non-diabetic controls. This suggests that GPX-002 can lead to transdifferentiation of alpha cells into a new population of beta-like cells that make insulin but still retain the capacity to produce glucagon.
In conclusion, the novel rAAV gene therapy research demonstrated that infusion of rAAV directly into the pancreatic duct of NHPs leads to transdifferentiation of alpha cells to beta-like cells with restoration of glucose homeostasis. IS, including steroids, is necessary for a number of months to prevent anti-viral immunity in NHPs.
Researchers are continuing preclinical studies of GPX-002 therapy in NHP models of both Type 1 and Type 2 diabetes to generate additional data, and current studies are evaluating viral efficacy after six months of IS.
About GPX-002
GPX-002, which has been exclusively licensed from the University of Pittsburgh, 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.
About Genprex, Inc.
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. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches.
Genprex’s oncology program utilizes its systemic, non-viral Oncoprex® Delivery System which encapsulates the gene-expressing plasmids using lipid-based nanoparticles in a lipoplex form. The resultant product is administered intravenously, where it is taken up by tumor cells that then express tumor suppressor proteins that were deficient in the tumor. The Company’s lead product candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid), is being evaluated in two clinical trials as a treatment for NSCLC and SCLC. Each of Genprex’s lung cancer clinical programs has received a Fast Track Designation from the FDA for the treatment of that patient population, and Genprex’s SCLC program has received an FDA Orphan Drug Designation.
Genprex’s diabetes gene therapy approach is comprised of a novel infusion process that uses an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, GPX-002 transforms 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 a similar approach for Type 2 diabetes, where autoimmunity is not at play, GPX-002 is believed to rejuvenate and replenish exhausted beta cells.
Interested investors and shareholders are encouraged to sign up for press releases and industry updates by visiting the Company Website, registering for Email Alerts and by following Genprex on Twitter, Facebook and LinkedIn.
SOURCE: Genprex
Post Views: 1,209
Latest Research Demonstrates Promising Improved Glucose Homeostasis by Reprogramming Alpha Cells
AUSTIN, TX, USA I June 24, 2025 I Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that its research collaborators presented positive preclinical research from studies of GPX-002, the Company’s diabetes gene therapy drug candidate, at the 2025 American Diabetes Association (ADA) 85th Scientific Session in Chicago.
“We are proud of the research presented at the ADA conference, which demonstrates that alpha cells in animal models of T1D have undergone transdifferentiation to beta-like cells after being transduced with GPX-002. In addition, the beta-like cells were still providing improved control of glucose levels after three months,” said Ryan Confer, President and Chief Executive Officer at Genprex. “We are very excited to continue evaluating GPX-002 as we further optimize how to control anti-viral immunity and move toward human clinical trials.”
The oral presentation details for the Genprex-supported abstract at the ADA 85th Scientific Sessions:
Title: Recombinant AAV-mediated Gene Therapy For The Treatment Of Streptozotocin-Induced Diabetes in Non-Human Primates
Type: Oral Presentation
Track: Immunology/Beta-Cell Replacement
Category: Exploring Alternative Strategies to Stem Cell-Derived Beta Cells and Insights from Autologous Islet Transplantation
Presentation Date: Monday, June 23, 2025
Presentation Time: 1:30 p.m. CT
Presenter: Hannah Rinehardt, MD, University of Pittsburgh Medical Center
The oral presentation discussed GPX-002, the diabetes gene therapy, which uses infusion of recombinant adeno-associated virus (rAAV) retrograde into the pancreatic duct to deliver the Pdx1 and MafA genes. The gene therapy converts alpha cells into beta-like cells that secrete insulin physiologically, reversing diabetes in mouse models, where immunosuppression was not necessary. Researchers evaluated the immune response to direct infusion of rAAV into the pancreatic duct of non-human primates (NHPs) with streptozotocin-induced diabetes and evaluated how to best manage immune responses against the virus capsid proteins.
Diabetes was induced with streptozotocin (STZ) in cynomolgus macaques, a type of NHP. NHPs received retrograde intraductal infusion of rAAV via laparotomy for precise delivery to the pancreas. rAAV capsids were chosen based on tropism for endocrine cells, and pre-existing neutralizing antibody titers (NAbs) were negative.
Expression of viral proteins occurs for a limited period of time after rAAV infection, since the infection doesn’t produce new AAV virus. One-month post-infusion, NHPs showed improved glucose tolerance and reduced insulin requirements. In the following months, using steroid-sparing regimens, increases in pancreatic B and T lymphocyte populations were noted on single cell RNA sequencing. Temporary immunosuppression (IS), using a combination of rituximab, rapamycin, and steroids for a 3-month course is largely effective at preventing anti-viral immunity. However, discontinuation of IS at 3 months post-infusion led to an immune response afterwards, indicating that IS in NHPs may need to be continued longer, and six months of IS in NHPs is now being evaluated.
When colocalization of insulin and glucagon is quantified, there was significantly elevated colocalization in treated islets compared to untreated diabetic and non-diabetic controls. This suggests that GPX-002 can lead to transdifferentiation of alpha cells into a new population of beta-like cells that make insulin but still retain the capacity to produce glucagon.
In conclusion, the novel rAAV gene therapy research demonstrated that infusion of rAAV directly into the pancreatic duct of NHPs leads to transdifferentiation of alpha cells to beta-like cells with restoration of glucose homeostasis. IS, including steroids, is necessary for a number of months to prevent anti-viral immunity in NHPs.
Researchers are continuing preclinical studies of GPX-002 therapy in NHP models of both Type 1 and Type 2 diabetes to generate additional data, and current studies are evaluating viral efficacy after six months of IS.
About GPX-002
GPX-002, which has been exclusively licensed from the University of Pittsburgh, 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.
About Genprex, Inc.
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. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches.
Genprex’s oncology program utilizes its systemic, non-viral Oncoprex® Delivery System which encapsulates the gene-expressing plasmids using lipid-based nanoparticles in a lipoplex form. The resultant product is administered intravenously, where it is taken up by tumor cells that then express tumor suppressor proteins that were deficient in the tumor. The Company’s lead product candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid), is being evaluated in two clinical trials as a treatment for NSCLC and SCLC. Each of Genprex’s lung cancer clinical programs has received a Fast Track Designation from the FDA for the treatment of that patient population, and Genprex’s SCLC program has received an FDA Orphan Drug Designation.
Genprex’s diabetes gene therapy approach is comprised of a novel infusion process that uses an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, GPX-002 transforms 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 a similar approach for Type 2 diabetes, where autoimmunity is not at play, GPX-002 is believed to rejuvenate and replenish exhausted beta cells.
Interested investors and shareholders are encouraged to sign up for press releases and industry updates by visiting the Company Website, registering for Email Alerts and by following Genprex on Twitter, Facebook and LinkedIn.
SOURCE: Genprex
Post Views: 1,209
