Directed Differentiation of Gene-Edited Pluripotent Stem Cells

Pluripotent stem cell-derived therapies can involve directed differentiation of pluripotent stem cells to specific cell lineages. In gene-edited cells, directed differentiation can be hindered by transgene-encoded proteins.

Our scientists developed a technology for differentiating gene-edited pluripotent stem cells comprising a transgene that uses small-molecule inhibitors of the transgene-encoded protein⁷.

⁷Klee, D., et al. Mol Ther, Vol 31, No 4S1, 2023.

Directed Differentiation of Gene-Edited Pluripotent Stem Cells is protected by a pending U.S. patent (with additional patents pending in other countries).

• High-efficiency differentiation of gene-edited pluripotent stem cells
• High-purity transgene-expressing pluripotent stem cell-derived cells
• Autologous and allogeneic engineered cell therapies (e.g., CAR-T, CAR-NK, gene-edited stem cell-derived therapies, etc.)

• Combine with Factor’s mRNA Cell Reprogramming technology to generate models of genetic disease, gene-corrected patient-specific cell therapies, and allogeneic (i.e., immuno-nonreactive or “stealth”) cell therapies, including allogeneic pluripotent stem cell-derived CAR-T and CAR-NK cell therapies for the treatment of cancer, and engineered mesenchymal stem cell (MSC) therapies for regenerative medicine, wound-healing, inflammatory and auto-immune diseases, and tumor-targeting applications