Genprex has selected a U.S.-based contract development and manufacturing organization to scale production of its investigational diabetes gene therapy, moving the program from research-grade manufacturing toward clinical and commercial-ready output.
The clinical-stage gene therapy company said the agreement will provide manufacturing infrastructure for its adeno-associated virus, or AAV-based, therapy in a current Good Manufacturing Practices-compliant facility. The CDMO will manufacture drug product for Genprex’s IND-enabling preclinical studies and potential future clinical trials.
Genprex did not identify the CDMO or disclose financial terms of the agreement.
The company said it has transferred its manufacturing process from an academic setting to the CDMO, a transition intended to support upcoming toxicology studies and eventual clinical evaluation. Genprex President and Chief Executive Officer Ryan Confer said the agreement is an important step in advancing the diabetes gene therapy program.
The manufacturing roadmap includes process optimization using newer AAV production technologies, increased dose availability through larger-capacity bioreactors, development and qualification of potency assays, and quality-control procedures for potency, stability and purity. Genprex said the work will be conducted under cGMP standards to support future regulatory submissions.
Genprex’s diabetes candidate uses an AAV vector to deliver the Pdx1 and MafA genes into the pancreas through the pancreatic duct. The company said it has incorporated technologies to optimize the plasmid construct, improve stability of expression and align its backbone with other plasmids used in AAV products.
The company said the candidate is being developed as a potential treatment for both Type 1 and Type 2 diabetes.
For Type 1 diabetes, Genprex’s approach is designed to transform pancreatic alpha cells into functional beta-like cells capable of producing insulin. The company said the cells may be distinct enough from native beta cells to potentially avoid immune-system attack. In preclinical Type 1 diabetes mouse models, Genprex said the candidate restored normal blood glucose levels for an extended period. In nonhuman primate models, it reduced insulin requirements, increased C-peptide levels and improved glucose tolerance.
For Type 2 diabetes, the company said the therapy is intended to replenish and rejuvenate exhausted insulin-producing beta cells. Genprex is also evaluating second-generation AAV and non-viral approaches, including lipid nanoparticle delivery that could potentially enable repeat dosing.