Genprex (NASDAQ: GNPX) announced promising preclinical data on GPX-002, its diabetes gene therapy candidate, presented at the 2025 American Diabetes Association (ADA) Scientific Session. The research highlights a second-generation approach using a non-viral lipid nanoparticle (LNP) delivery system, aiming for repeat dosing.
“This is a significant step forward, showing our ability to use LNPs with nucleic acid payloads to transfect cells in the islets of Langerhans via direct infusion into the biliary/pancreatic duct,” said Ryan Confer, Genprex CEO. “We believe we’re the first to do this in the pancreatic duct with an LNP, providing proof of concept for a next-generation approach that could enable repeat dosing, crucial for Type 1 Diabetes treatment.”
Studies evaluated LNPs with patented LipexSil® lipids and mRNA payloads, showing high transfection efficiency in isolated mouse Islets of Langerhans. An LNP using ALX-184 with a Luciferase mRNA payload efficiently transfected pancreatic cells and crossed the basement membrane.
Dr. Mark Berger, Genprex CMO, emphasized the groundbreaking nature of the LNP research, noting the selection of a less toxic lipid and demonstrating 100 times more luciferase activity in the pancreas compared to other organs, proving transduction specificity. The LNP also crossed the basement membrane and penetrated islets, paving the way for further studies to improve diabetes treatments.
This research, alongside ongoing AAV construct studies, focuses on optimizing next-generation constructs. Non-viral delivery offers benefits like re-dosing potential and potentially eliminating immunosuppression.
About GPX-002
GPX-002, licensed from the University of Pittsburgh, is being developed for Type 1 (T1D) and Type 2 (T2D) diabetes. The core AAV-based approach delivers Pdx1 and MafA genes directly to the pancreatic duct. For T1D, it aims to transform alpha cells into functional, immune-evading beta-like cells. Preclinical studies show it restored normal blood glucose in T1D mouse models. For T2D, GPX-002 is believed to rejuvenate exhausted beta cells. The LNP research explores an alternative delivery system for repeat administration.
Source:
https://www.biospace.com/press-releases/genprex-collaborators-present-research-on-non-viral-approach-to-diabetes-gene-therapy-using-lipid-nanoparticle-delivery-system-at-the-2025-american-diabetes-association-85th-scientific-sessions
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PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
