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CyT49
- Development stage
- Unknown
- Lead developer
- Vertex
- Modality
- Stem Cell Therapies → Cell Therapies
- Administration
- Subcutaneous, Intraperitoneal
01
Overview
CyT49 is a proprietary human embryonic stem cell (hESC) line that serves as the foundational biological platform for a suite of regenerative medicine therapies developed by ViaCyte (now a subsidiary of Vertex Pharmaceuticals). The cell line is utilized to produce pancreatic endoderm cells (PEC-01), which are designed for implantation into patients with insulin-requiring diabetes, particularly Type 1 Diabetes (T1D). Once transplanted, these progenitor cells mature in vivo into functional, glucose-responsive, insulin-secreting beta cells and other islet endocrine cells, effectively replacing the endocrine function of a damaged pancreas. CyT49 is also the substrate for next-generation "immune-evasive" therapies, such as VCTX210 and VCTX211, which employ CRISPR/Cas9 gene editing to knock out the B2M gene and overexpress PD-L1. These modifications are intended to protect the cells from host immune rejection, potentially eliminating the need for chronic immunosuppressive therapy.
- Other names
- human embryonic stem cell line CyT49hESC line CyT49ViaCyte stem cell line
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