Andelyn to Scale Manufacturing for Genprex’s Diabetes Gene Therapy Programme

Andelyn Biosciences has been selected by Genprex to support the manufacturing scale-up of its experimental gene therapy programme for diabetes, marking an important step as the company prepares the programme for IND-enabling preclinical studies and potential clinical development.

The collaboration will focus on Genprex’s adeno-associated virus (AAV)-based candidate, which is designed to deliver the Pdx1 and MafA genes directly to the pancreas via the pancreatic duct. The approach is being investigated as a potential treatment for both Type 1 and Type 2 diabetes.

As part of the agreement, Andelyn will work with Genprex to transition the programme towards a scalable manufacturing process, with activities spanning process optimisation, scale-up, analytical development and cGMP manufacturing.

Building a scalable AAV manufacturing process

The manufacturing programme will leverage Andelyn’s Curator Cell and Gene Therapy Platform and its experience in AAV process development and manufacturing.

Planned activities include process optimisation, technology transfer, analytical qualification, potency assay development and drug product manufacturing within a cGMP-compliant facility.

The move towards a scalable cGMP process represents a key development milestone for Genprex as it works to progress the therapy from preclinical development towards clinical evaluation.

Targeting insulin production in the pancreas

Genprex’s approach is based on using gene therapy to modify pancreatic cells involved in insulin production.

For Type 1 diabetes, the company is investigating whether delivery of Pdx1 and MafA can transform pancreatic alpha cells into functional beta-like cells capable of producing insulin. Genprex has reported preclinical results in mouse models in which the approach restored normal blood glucose levels for an extended period.

The company has also reported findings from nonhuman primate studies indicating 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 driver of disease, Genprex is investigating whether the same approach could help replenish or rejuvenate exhausted insulin-producing beta cells. The company has reported preclinical activity in both mouse and nonhuman primate models.

Preparing for the clinic

For cell and gene therapy developers, moving from research-scale production to a robust, reproducible cGMP manufacturing process is an important part of preparing an investigational therapy for clinical development.

Genprex's selection of Andelyn therefore provides the programme with manufacturing capabilities intended to support its next development stages, including IND-enabling studies and eventual clinical trials.

“Andelyn’s experience in AAV development and cGMP manufacturing will help us advance the manufacturing process as we work to move this program toward the clinic,” said Ryan Confer, President and CEO of Genprex.

The companies' collaboration is expected to support Genprex as it establishes the manufacturing foundation required to progress its diabetes gene therapy programme through preclinical development and towards first-in-human evaluation.