Andelyn Scales AAV Manufacturing for Diabetes Gene Therapy

Columbus, Ohio, September 1, 2026

AAV Manufacturing Moves Toward Clinical Development

Under the agreement, Andelyn will support the transition of Genprex’s diabetes gene therapy candidate through process optimization, manufacturing scale-up, IND-enabling production and clinical-stage manufacturing. The planned activities include analytical qualification, potency assay development and drug product manufacturing within a cGMP-compliant facility. This manufacturing transition represents an important development step for an advanced therapy because production processes established during research must be transformed into robust, reproducible and appropriately controlled processes capable of supporting regulatory and clinical requirements. Andelyn said its work will leverage the company’s Curator® Cell and Gene Therapy Platform, together with expertise in AAV process development, technology transfer, analytical development and cGMP manufacturing. The collaboration is focused not only on increasing manufacturing scale but also on establishing the technical and quality foundation needed for Genprex’s upcoming development milestones. Manufacturing readiness is a critical component of gene therapy development, particularly as programs progress from laboratory research toward IND-enabling studies and first-in-human clinical evaluation. The planned process optimization and analytical activities are expected to help characterize the product and establish manufacturing controls suitable for continued development.

Pdx1 and MafA Gene Therapy Targets Diabetes

Genprex’s candidate uses an AAV vector carrying Pdx1 and MafA genes, administered directly into the pancreas through the pancreatic duct. According to Genprex, the same general approach is being evaluated for both Type 1 and Type 2 diabetes. In Type 1 diabetes, the strategy is designed to transform pancreatic alpha cells into functional beta-like cells capable of producing insulin, potentially providing a new source of insulin-producing cells. In preclinical Type 1 diabetes mouse models, Genprex has reported restoration of normal blood glucose levels for an extended period. Studies in nonhuman primates have also reportedly shown reduced insulin requirements, increased C-peptide levels, and improved glucose tolerance. For Type 2 diabetes, where autoimmune destruction of beta cells is not the primary disease mechanism, Genprex is investigating whether the gene therapy approach could help replenish or rejuvenate exhausted insulin-producing beta cells. The company has reported preclinical activity in both mouse and nonhuman primate models. However, these findings remain preclinical, and the therapeutic potential of the approach will require evaluation in appropriately designed clinical studies.

cGMP Platform Supports IND-Enabling Manufacturing

Andelyn brings extensive experience in cell and gene therapy manufacturing, with capabilities spanning viral-vector development, analytical development, process optimization and cGMP production. The company states that its experience includes production of cGMP material for more than 500 clinical batches and 85 global clinical trials. Its Curator platform is designed to support gene therapy programs through scalable process development and manufacturing. For Genprex, this expertise is intended to establish a manufacturing pathway that can support the diabetes program as it advances toward IND-enabling studies and future clinical trials. The selection of Andelyn follows Genprex’s broader efforts to advance its diabetes gene therapy toward clinical development. Genprex describes the program as a novel approach using Pdx1/MafA gene delivery to address insulin-producing cell function in both major forms of diabetes. Earlier 2026 company updates reported additional preclinical research evaluating the technology in Type 2 diabetes models. The new manufacturing collaboration therefore represents a significant development and CMC milestone rather than a clinical efficacy result. By moving toward optimized processes, qualified analytical methods, potency testing and cGMP drug-product manufacturing, Genprex is working to establish the manufacturing infrastructure required for regulatory advancement. The next major milestones will include completion of the necessary IND-enabling work and preparation for clinical evaluation, which will determine whether the promising preclinical gene therapy strategy can ultimately demonstrate safety and therapeutic benefit in people with diabetes.

Source: Andelyn Biosciences press release