ViaCyte, a stem cell therapy company focused on developing treatments for diabetes, has announced the closing of a $27 million financing round with participation from Bain Capital Life Sciences to further advance the company’s development of multiple drug candidates in an effort to provide potential new therapies for diabetes patients. According to public information, the company has now closed over $200 million in financing.
ViaCyte, which has two proprietary technology platforms, a stem cell expansion and differentiation platform and Encaptra, an implantable device for cell delivery, is developing its PEC family of drugs by differentiating pluripotent stem cells into islet precursors (PEC-01 cells). Currently, two of its three PEC family drug candidates are in clinical development.
The company’s PEC-Direct (VC-02) therapy was developed to allow blood vessels to enter the device and interact directly with the implanted PEC-01 cells, with the aim of achieving a solid implantation. However, because the implanted cells are not hidden by the immune system, patients will require immunosuppressive drugs to protect the cells and are therefore only indicated for high-risk type 1 diabetes patients. Clinical data show that its implanted cells are capable of producing circulating C-peptides (biomarkers of insulin) in patients with type 1 diabetes. The company is also currently developing its treatment for type 1 diabetic patients with hypoglycemia, blood glucose instability, and/or recurrent severe hypoglycemic events.
PEC-Encap (VC-01) is an islet precursor placed in the Encaptra delivery system and administered by subcutaneous implantation. the Encaptra system typically prevents immune rejection and immunosensitization, effectively protecting transplanted cells from the patient’s adaptive immune system. Once implanted, PEC-01 cells are transformed into endocrine cells and secrete insulin and other hormones in a regulated manner to control blood glucose levels, just like endocrine cells in the pancreas of a healthy person.
In collaboration with CRISPR Therapeutics, ViaCyte has made special designs based on its proprietary pluripotent stem cell line, CyT49, that allow it to eliminate the disruption of the patient’s immune system and thus obviate the need for immunosuppression. They have used gene editing technology to knock out a microglobulin gene in the CyT49 pluripotent stem cell line that is associated with T-cell attack, thereby protecting islet cells from rejection. These stem cell lines are being used in the development of PEC-QT (VCTX210), which has the potential to be a potential therapy for the treatment of patients with insulin-requiring type 1 and type 2 diabetes.
“We thank these outstanding investors, as well as our clinical trial participants, for their continued support. We will continue to offer potential new therapies for patients with diabetes who require insulin therapy,” said Paul Laikind, Ph.D., CEO, and President of ViaCyte. “Looking forward, we will continue to optimize the efficacy of PEC-Direct and PEC-Encap, and with our partner CRISPR Therapeutics, we will work to develop PEC-QT to treat patients in need.”