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PEC210A
- Development stage
- Unknown
- Lead developer
- CRISPR Therapeutics
- Modality
- Cell Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Implantation (subcutaneous)
01
Overview
PEC210A is a genetically engineered **allogeneic pancreatic endoderm cell therapy** derived from human embryonic stem cells, designed for the treatment of type 1 diabetes. PEC210A is the cell component in the VCTX210A product and has undergone CRISPR/Cas9 editing to enhance immune evasion: *B2M* knockout to abrogate MHC class I, overexpression of PD-L1 and HLA-E to suppress cytotoxic T cell and NK cell activity, and *TXNIP* knockout to increase resistance to oxidative and endoplasmic reticulum stress. These cells are encapsulated in a device ("PEC-Direct" macroencapsulation device) that permits vascularization and nutrient exchange while exposing cells to the host immune system, aiming to restore insulin production in type 1 diabetes patients without the need for systemic immunosuppression. PEC210A is developed and manufactured as part of the VCTX210A product by ViaCyte (a subsidiary of Vertex Pharmaceuticals) in collaboration with CRISPR Therapeutics[9][10][11][12][8][7].
- Other names
- PEC210APEC-210APEC 210A
02
Targets
CD274 (Programmed cell death protein 1 ligand 1)TXNIP (Thioredoxin-interacting protein)HLA-E (Human leukocyte antigen-e)
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