Genprex, a clinical-stage gene therapy company developing treatments for cancer and diabetes, has entered into a development and manufacturing agreement with a US-based clinical and commercial contract development and manufacturing organisation (CDMO) to scale up manufacturing of its diabetes gene therapy programme.

The partnership is intended to facilitate the transition of Genprex’s therapeutic candidate from research-grade production to clinical- and commercial-ready manufacturing. Under the agreement, the CDMO will manufacture and supply the drug product in a current Good Manufacturing Practices (cGMP)-compliant facility for use in IND-enabling preclinical studies and future clinical trials.

According to Genprex, the collaboration provides access to specialised infrastructure and expertise required to manufacture its Adeno-Associated Virus (AAV)-based therapy at increased scale while maintaining stringent quality standards.

Ryan Confer, President and Chief Executive Officer, Genprex, said, “This strategic partnership represents a significant advancement for our diabetes gene therapy clinical programme. 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.”

The company said the collaboration is expected to support the programme’s progression towards clinical evaluation, including toxicology studies focused on establishing the safety and efficacy profile required for future development.

The manufacturing programme will focus on process optimisation, scale-up, analytical qualification, potency assay development and cGMP compliance. Genprex plans to apply advanced AAV production technologies to improve manufacturing efficiency and use larger-capacity bioreactors to increase drug product availability in preparation for potential multi-centre clinical trials.

The company will also implement analytical and quality-control protocols to assess AAV potency, stability and purity, while maintaining compliance with cGMP requirements to support future regulatory submissions.

Genprex’s diabetes gene therapy candidate is designed to deliver the Pdx1 and MafA genes into the pancreas through the pancreatic duct using an AAV vector. The company has incorporated advanced technologies to optimise the plasmid construct, improve expression stability and align the plasmid backbone with other plasmids used in AAV-based products. The resulting plasmid has been cloned, purified and manufactured and is currently being used in AAV production.

Beyond the current AAV approach, Genprex is working with CDMOs and research partners to optimise its constructs and assess second-generation approaches, including alternative AAV and non-viral delivery technologies. The company is also collaborating with a CDMO to investigate lipid nanoparticle-based, non-viral delivery of its diabetes gene therapy candidate, which could potentially enable re-dosing and allow treatment to be optimised over time.

The candidate is being developed as a potential treatment for both Type 1 diabetes (T1D) and Type 2 diabetes (T2D). For T1D, the approach is designed to transform pancreatic alpha cells into functional beta-like cells capable of producing insulin while potentially reducing recognition by the immune system.

In preclinical T1D mouse models, Genprex reported that the candidate restored normal blood glucose levels for an extended period. Studies in T1D non-human primate (NHP) models also demonstrated reduced insulin requirements, increased C-peptide levels and improved glucose tolerance.

For T2D, where autoimmune destruction of beta cells is not the primary mechanism, the company’s approach is intended to replenish and rejuvenate exhausted insulin-producing beta cells. Genprex has reported activity supporting this approach in both mouse and NHP models.

The latest manufacturing agreement marks a step towards establishing the production capabilities required for Genprex’s diabetes gene therapy programme to advance through IND-enabling studies and potentially into clinical development.