ViaCyte’s Breakthrough Technologies

Addressing unmet medical needs with innovative cell replacement therapies

Our regenerative medicine platforms represent two major technological advances: stem cell-derived cell replacement therapies and the Encaptra® system for cell encapsulation and implantation. We have the opportunity to use each technology individually or together to address critical human diseases and disorders that can potentially be treated by replacing lost or malfunctioning cells or proteins.

Our approach to insulin-requiring diabetes

ViaCyte’s history of leadership and firsts in developing stem cell-derived cell replacement therapies for type 1 diabetes.

We are focused on developing a functional cure for type 1 diabetes. ViaCyte leads the industry in developing islet cell replacement therapies for insulin-requiring diabetes. As seen in the scientific published literature, the Company’s scientists were the first to describe directed differentiation of human pluripotent stem cells into insulin-expressing pancreatic cells and the first to demonstrate the ability of these cells to faithfully secrete insulin in response to increased blood glucose in pre-clinical models. ViaCyte has shown that these cells differentiate in vivo, in both preclinical and clinical studies, to all the constituent cell types of the human pancreatic islet.

ViaCyte’s approach seeks to address the hurdles of donor islet transplants

Decades of clinical data with donor islet transplants indicate that beta cell replacement approaches may offer curative benefit to patients with type 1 diabetes. While donor islet transplants can be highly effective, they are not widely available because of a severe lack of suitable donor organs and the requirement for immune suppression.

To overcome these hurdles, ViaCyte has pioneered the approach of carefully and fully differentiating pluripotent stem cells into pancreatic islet precursor cells (PEC-01 cells) and delivering them to patients. Since PEC-01 cells are made from a pluripotent stem cell line (CyT49), there is a potentially unlimited supply available for manufacturing ViaCyte’s product candidates.

At ViaCyte, our mission is to profoundly improve the lives of people with type 1 diabetes. To accomplish that, we have become leaders and pioneers in many areas, breaking new ground in stem cell research and medical device engineering, and developing brand new methods of cell therapy scaling and manufacturing.

ViaCyte has multiple therapeutic approaches to propel clinical development

For PEC-Encap, PEC-01 cells are loaded into the Encaptra system, which is implanted under the patient’s skin (subcutaneously). The Encaptra system is designed to encapsulate and protect the cells from the immune system. After implantation, PEC-01 cells are designed to mature into all the types of cells in the endocrine pancreas. Once matured, the implanted cells have the potential to secrete insulin and other hormones in order to respond to and control blood glucose (sugar) levels.

For PEC-Direct, the PEC-01 cells are contained within a device designed to allow direct vascularization. Because the implanted cells are not hidden from the immune system, patients who receive PEC-Direct must take immunosuppressants, making it suitable only for people with high-risk type 1 diabetes.

For the PEC-QT program, ViaCyte is working with CRISPR Therapeutics to use gene-editing technology to create an immune-evasive version of ViaCyte’s CyT49 pluripotent stem cell line. Cells differentiated from this line may avoid rejection by the patient’s immune system, thus potentially eliminating the need for immunosuppressants and creating a next-generation potential functional cure for all insulin-requiring diabetes.

With breakthrough technology, clinical stage product candidates, an extensive intellectual property estate, and a strong and dedicated team, ViaCyte has all the pieces to advance transformative cell replacement therapies for diabetes.