In type 1 diabetes, the insulin-producing beta cells of the endocrine pancreas are destroyed by a person’s own immune system in a disease process known as autoimmunity. Consequently, those with type 1 diabetes are unable to produce insulin, which is necessary for control of blood glucose, and ultimately, for life. While pharmaceutical insulin provides life-saving relief, numerous risks and complications exist for those contending with this disease. A large body of research indicates that replacing the lost beta cells of the endocrine pancreas would represent a substantially more effective approach to treating type 1 diabetes.
To address this need, ViaCyte is developing a cell replacement therapy for type 1 diabetes. The proposed product is comprised of human pancreatic progenitor cells, known as PEC-01™ cells, loaded into and implanted within a flat encapsulation device or packet, known as the Encaptra® drug delivery system. PEC-01 cells are manufactured in the laboratory from human embryonic stem cells using a proprietary process that was developed at ViaCyte. The Encaptra device is made of biocompatible materials and is designed to retain PEC-01 cells in a specific location in the body, and protect them from a patient’s immune system. Together, PEC-01 cells within the Encaptra device are known as the VC-01™ combination product candidate, and animal studies have shown that following implant, the cells mature into functional human pancreatic endocrine cells (including insulin producing cells) that respond to changes in blood glucose appropriately, including the release of insulin into the circulation when blood glucose levels are increased.
The Strategic Partnership Award provides funding to support the preparation and launch of clinical testing of the VC-01 product candidate. The first year of the award was to support pre-clinical work including the preparation and filing of regulatory documents including, importantly, an Investigational New Drug (IND) application with the FDA, to allow testing in human subjects to commence.
The team made great progress in the first year of the award and achieved all planned milestones in a timely fashion. Specifically, the group was able to complete the necessary animal safety and efficacy studies (performed under Good Laboratory Practices) that are prerequisite to human testing, obtain a license from the California Food and Drug Branch to allow product manufacturing to proceed, and manufacture PEC-01 cells (under Good Manufacturing Practices) and Encaptra devices (under Quality Systems Regulations) for the Phase 1/2 first-in-human clinical trial. Moreover, the group established and finalized materials and methods to load PEC-01 cells into Encaptra devices and prepare the sealed VC-01 combination product for transport to the point of service. The group also established storage conditions for the VC-01 product consistent with the logistics of the clinical trial. Lastly, the clinical plan and protocol were completed and the IND was submitted. Within 30 days the IND was allowed by the FDA, and the first clinical site was prepared to launch the trial.