CIRM Grants ViaCyte $1.4M to Create Immune-Evasive Pluripotent Stem Cell Lines



SAN DIEGO, Dec. 15, 2017 /PRNewswire/ -- ViaCyte, Inc., a privately-held regenerative medicine company, today announced that the California Institute for Regenerative Medicine (CIRM) approved a grant of $1.4 million to support the initial development of immune-evasive pluripotent stem cell lines. The focus of the project will be to genetically engineer the Company's CyT49 pluripotent stem cell line. ViaCyte's proprietary CyT49 cell line is well characterized and has been used to manufacture cell replacement product candidates that have been reviewed and allowed for use in clinical trials by multiple regulatory authorities.

One of the main challenges in developing an 'off-the-shelf' cell therapy is the potential for immune rejection of implanted cells. Genetic engineering of a pluripotent stem cell line may make it possible to generate cell therapies from a single source that will not be rejected by the immune system. For diabetes, pluripotent stem cells have the potential of providing an unlimited supply of differentiated cells to overcome the current limitations of donor islet transplants, which have been shown to be highly effective in stabilizing blood glucose and providing insulin-independence in these patients but require immunosuppression. Delivering immune-evasive pancreatic progenitor cells to patients with type 1 diabetes may provide the benefits of islet transplant without the need for pharmacological immune suppression.

"The universal donor pluripotent stem cell line will represent a tremendous asset to ViaCyte, and the regenerative medicine field in general, and we are grateful to CIRM for their continued support to advance this new approach," said Kevin D'Amour, PhD, Vice President of Research and Chief Scientific Officer of ViaCyte. "Because all allogeneic cell therapies require some strategy to address patient immunity and potential rejection, and pluripotent stem cells can be made into any cell type in the body, we believe that an immune-evasive CyT49 line may potentially be used to manufacture not only replacement glucose-responsive insulin-producing cells for diabetes, but also cell therapies for numerous other unmet medical needs."

"ViaCyte is focused on the development of stem cell-derived cell replacement therapies to provide better treatment options for patients with diabetes and we are pleased to partner with them to pursue this innovative program," said Maria Millan, MD, President and CEO of CIRM. "Development of an immune-evasive cell therapy would increase the chances of engraftment and durable effect of a cell replacement therapy for diabetes. CIRM is currently funding ViaCyte's clinical stage program and is pleased to now fund this earlier stage research as it may lead to next generation versions of their therapeutic platform. CIRM's mission is to accelerate the delivery of stem cell-based treatments to patients with serious life-threatening conditions by funding research at all stages - discovery, translational, and clinical research."

Pluripotent stem cells have two important properties that make them extremely promising for biomedical cell therapy products. First, they have an effectively unlimited capacity for self-renewal and thus can be manufactured at large industrial scale. Second, they have the potential to be differentiated into any tissue in the body, provided the appropriate 'recipe' for directed differentiation is figured out. ViaCyte has spent the last decade developing the recipe for production of human islets; establishing the know-how, methods, controls, and protocols to generate large quantities of replacement pancreatic endocrine cells reproducibly under GMP conditions that could be used as a functional cure for type 1 diabetes. In parallel, ViaCyte has established a robust patent estate to protect the intellectual property assets of stem cell propagation and differentiation, and delivery of the cell replacement therapies. Immune-evasive pluripotent cells would further extend the many possible applications of this technology.

About ViaCyte

ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte's product candidates are based on the derivation of pancreatic progenitor cells from stem cells, which are then implanted in durable and retrievable cell delivery devices. Once implanted and matured, these cells are designed to secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two product candidates in clinical-stage development. The PEC-Direct™ product candidate delivers the pancreatic progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or recurrent severe hypoglycemic episodes. The PEC-Encap™ (also known as VC-01) product candidate delivers the same pancreatic progenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is headquartered in San Diego, California. The Company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF.

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