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NY-ESO-1 CD4-TCR-transduced CD34+ hematopoietic stem cells
- Development stage
- Phase 1
- Lead developer
- University of California, Los Angeles
- Modality
- TCR-Engineered T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Stem Cell Therapies → Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
NY-ESO-1 CD4-TCR-transduced CD34+ hematopoietic stem cells represent an investigative ex vivo gene and cell therapy designed for the treatment of NY-ESO-1-expressing malignancies, such as melanoma and synovial sarcoma. The therapeutic process involves the isolation of a patient's autologous CD34+ hematopoietic stem and progenitor cells (HSPCs), which are genetically modified using a lentiviral vector to express a T-cell receptor (TCR) specific for the cancer-testis antigen NY-ESO-1. This specific version utilizes a TCR restricted by MHC Class II (HLA-DP4), intended to generate a self-renewing population of antigen-specific CD4+ helper T cells. Once infused back into the patient following conditioning, these engineered HSPCs engraft in the bone marrow and provide a continuous, lifelong supply of NY-ESO-1-specific T cells. This approach aims to overcome the limitations of conventional adoptive T-cell transfer, such as T-cell exhaustion and limited persistence, by establishing a durable and regenerative anti-tumor immune system.
- Other names
- NY-ESO-1-specific CD4+ TCR-engineered hematopoietic stem cellsNY-ESO-1 TCR-transduced CD34+ HSPCsNY-ESO-1 CD4-TCR CD34+ cells
02
Targets
pMHC-II (Peptide-major histocompatibility complex class II complex)
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