Genprex has entered into a development and manufacturing agreement with an undisclosed, US-based clinical and commercial contract development and manufacturing organization (CDMO) to scale up manufacturing of its diabetes gene therapy candidate, the company announced August 18, 2026.¹ The collaboration is intended to transition the adeno-associated virus (AAV)-based therapy from research-grade production to clinical- and commercial-ready output in a current good manufacturing practice (CGMP)-compliant facility.
Key facts
- Drug: GPX-002 (Pdx1/MafA gene therapy; Genprex)
- Class: AAV-vector gene therapy (pancreatic ductal delivery)
- Indications studied: T1D and T2D
- Milestone: CDMO development and manufacturing agreement, August 18, 2026
- Manufacturing priorities: Process optimization, scale-up, potency assay development, CGMP compliance
- Key T2D data: Reversed hyperglycemia in mice within 4 weeks (ASGCT 2026)
- Key T1D data: Decreased insulin needs, increased C-peptide, improved glucose tolerance (NHP models)
- Regulatory status: Preclinical; IND-enabling studies planned
- Pipeline context: Lead oncology candidate quaratusugene ozeplasmid in 2 trials (both FDA fast track)
- Geography: US-based CDMO; company headquartered in Austin, Texas
"This strategic partnership represents a significant advancement for our diabetes gene therapy clinical program," said Ryan Confer, president and CEO, Genprex, in a company press release.¹ "We have transferred the manufacturing process for our diabetes product candidate from the academic setting, where it was previously manufactured, into a reputable CGMP compliant CDMO."
What will the CDMO manufacture, and what are the technical priorities?
The CDMO will manufacture and supply drug product for Genprex's investiational new drug (IND)-enabling preclinical studies and future clinical trials. The manufacturing roadmap includes 4 technical priorities: process optimization using AAV production technologies to increase efficiency; scale-up using capacity bioreactors to increase dose availability ahead of multicenter trials; analytical qualification and development of a potency assay to ensure AAV potency, stability, and purity; and CGMP compliance to support future regulatory submissions. Genprex said this collaboration marks the transition of its diabetes candidate's manufacturing process from an academic setting to a CDMO, following a broader technology transfer effort the company outlined in January 2026.²
How does the diabetes gene therapy work?
Genprex's diabetes candidate, GPX-002, delivers the Pdx1 and MafA genes into the pancreas via the pancreatic duct using an AAV vector. In type 1 diabetes (T1D), the therapy is designed to transform alpha cells into functional beta-like cells capable of producing insulin while potentially evading immune attack. In preclinical non-human primate models, it decreased insulin requirements, increased C-peptide levels, and improved glucose tolerance.¹
In type 2 diabetes (T2D), where autoimmunity is not a factor, the same approach is designed to replenish and rejuvenate exhausted beta cells. In mouse models presented at the 2026 American Society of Gene and Cell Therapy Annual Meeting, the therapy reversed hyperglycemia within 4 weeks and improved glucose-stimulated insulin secretion to levels comparable with non-diabetic controls.³
What's next for Genprex's broader pipeline?
Genprex is also evaluating second-generation approaches for the diabetes program, including alternative AAV constructs and a non-viral lipid nanoparticle delivery method that could allow for potential re-dosing of patients. The company's lead oncology candidate, quaratusugene ozeplasmid (Reqorsa), is being evaluated in 2 clinical trials for non-small cell and small cell lung cancer, both of which hold FDA fast track designation, with the small cell lung cancer program also holding FDA orphan drug designation.¹
What are the limitations?
Genprex did not disclose the name of the CDMO nor the financial terms of the agreement. The diabetes program remains in the preclinical stage, and no clinical trials in humans have yet begun.
References
- Genprex. Genprex selects leading clinical and commercial contract development and manufacturing organization for manufacturing scale up of diabetes gene therapy. Press release. Published August 18, 2026. Accessed August 18, 2026. https://www.genprex.com/2026/08/18/genprex-selects-leading-clinical-and-commercial-contract-development-and-manufacturing-organization-for-manufacturing-scale-up-of-diabetes-gene-therapy/
- Genprex. Genprex provides clinical update on diabetes gene therapy program. Press release. Published January 7, 2026. Accessed August 18, 2026. https://www.genprex.com/2026/01/07/genprex-provides-clinical-update-on-diabetes-gene-therapy-program/
- Genprex. Genprex collaborators present positive preclinical data on diabetes gene therapy for Type 2 diabetes at the 2026 American Society of Gene and Cell Therapy Annual Meeting. Press release. Published May 14, 2026. Accessed August 18, 2026. https://www.genprex.com/2026/05/14/genprex-collaborators-present-positive-preclinical-data-on-diabetes-gene-therapy-for-type-2-diabetes-at-the-2026-american-society-of-gene-and-cell-therapy-annual-meeting/
