Genprex Collaborators to Present Positive Preclinical Data on Diabetes Gene Therapy at the ASGCT 28th Annual Meeting

Core Viewpoint - Genprex, Inc. has announced the presentation of positive preclinical data for its diabetes gene therapy candidate GPX-002 at the upcoming ASGCT Annual Meeting, highlighting the potential of gene therapy as a curative treatment for diabetes [1][2]. Group 1: Research and Development - The oral presentation will focus on the immune modulation and reprogramming of alpha cells to beta-like cells in a diabetic non-human primate model, showcasing the effectiveness of the rAAV gene therapy approach [3]. - The study demonstrated that retrograde intraductal infusion of rAAV6 can convert alpha cells into insulin-secreting beta-like cells, reversing diabetes in mouse models [4]. - One month post-infusion, non-human primates exhibited improved glucose tolerance and reduced insulin requirements, indicating the therapy's durable effects [5]. Group 2: Immune Response Management - The research evaluated the immune response to direct infusion of rAAV into the pancreatic duct and how to manage potential anti-viral immunity, which can hinder gene therapy success [4][5]. - Temporary immunosuppression using a combination of rituximab, rapamycin, and steroids was largely effective in preventing anti-viral immune responses, allowing sustained therapeutic effects [6]. Group 3: Product Overview - GPX-002 is being developed for both Type 1 and Type 2 diabetes, utilizing an AAV vector to deliver Pdx1 and MafA genes directly into the pancreatic duct [7]. - In Type 1 diabetes, GPX-002 aims to transform alpha cells into functional beta-like cells, while in Type 2 diabetes, it is believed to rejuvenate exhausted beta cells [7]. Group 4: Company Background - Genprex, Inc. is a clinical-stage gene therapy company focused on developing therapies for cancer and diabetes, collaborating with leading institutions to advance its drug candidates [8].