Andelyn Biosciences has been selected by Genprex Inc. to support manufacturing scale-up for an adeno-associated virus-based gene therapy program being developed for Type 1 and Type 2 diabetes.
Under the agreement, Andelyn will help transition Genprex’s candidate from research-stage manufacturing through process optimization, scale-up and manufacturing activities intended to support investigational new drug-enabling studies and future clinical trials.
Genprex’s therapy is designed to deliver the Pdx1 and MafA genes directly to the pancreas through the pancreatic duct using an AAV vector. The program is intended to address diabetes by altering or restoring insulin-producing function in pancreatic cells.
For Type 1 diabetes, Genprex is developing the candidate to convert pancreatic alpha cells into functional beta-like cells that can produce insulin. The company has reported preclinical results from mouse models showing normal blood-glucose levels were restored for an extended period. In nonhuman primate models, Genprex reported reduced insulin requirements, higher C-peptide levels and improved glucose tolerance.
In Type 2 diabetes, Genprex is evaluating whether the treatment could replenish and rejuvenate exhausted insulin-producing beta cells. Unlike Type 1 diabetes, Type 2 diabetes is not primarily driven by autoimmunity. The company said it has observed preclinical activity in mouse and nonhuman primate models for this use as well.
Andelyn will use its Curator Cell and Gene Therapy Platform to perform AAV process development, technology transfer, analytical development and current good manufacturing practice, or cGMP, manufacturing. Planned activities include process optimization, scale-up, analytical qualification, potency-assay development and drug-product manufacturing in a cGMP-compliant facility.
The shift from academic or research-grade production to a scalable cGMP manufacturing process is a central step before an advanced therapy can enter clinical testing. The work is intended to establish a manufacturing foundation for Genprex’s planned preclinical and clinical milestones.
Matt Niloff, chief commercial officer at Andelyn, said the company would provide technical, operational and quality support as Genprex prepares the program for later development stages.
Ryan Confer, Genprex’s president and chief executive, said the selection of Andelyn will help prepare the therapy for IND-enabling studies and eventual clinical evaluation.
The agreement does not disclose financial terms, a development timeline or a projected date for Genprex to submit an investigational new drug application.