Progress on ViaCyte’s novel encapsulated cell therapy


In February, JDRF announced it is providing additional milestone-based funding for the continued development of ViaCyte’s VC-01™ encapsulated cell therapy product candidate for the treatment of T1D. JDRF will fund up to $7 million to help ensure a rapid transition of the project into the clinical phase of development once ViaCyte’s investigational new drug application (IND) is filed with and accepted by the U.S. Food and Drug Administration (FDA). This commitment builds on JDRF’s previous support of ViaCyte’s preclinical development program focused on collecting the necessary animal safety and efficacy data to support introduction into clinical testing. ViaCyte’s innovative VC-01 product candidate is a cell replacement therapy that could transform the way individuals with T1D manage their disease by supplying an alternative source of insulin-producing cells with the potential to free individuals from a dependence on external insulin use. The product candidate uses pancreatic progenitor cells derived from a stem cell line, called PEC-01™ cells, which are encapsulated inside a semi-permeable device called the Encaptra® drug delivery system. Both the cells and the device are ViaCyte proprietary technologies. The resulting VC-01 product candidate is designed to be inserted under the skin where, after maturation of the PEC-01 cells into islet-like structures including beta cells, they are expected to produce insulin and other pancreatic hormones in response to blood glucose levels, similar in manner to that of normal islets in the pancreas. ViaCyte is planning to file an IND with the FDA soon to support initiation of clinical evaluation of the VC-01 product candidate before the end of 2014. The primary purpose of the first human study will be to establish that the product candidate is safe and well tolerated; however, efficacy will also be assessed. After initial safety is demonstrated in the first group of participants, ViaCyte plans to expand the trial to multiple clinical sites in the United States and Canada.