Rapid non-viral platform for stable stem cell engineering

Researcher:

Categories:

Therapeutics & Diagnostics

Technology:

Engineering human pluripotent stem cells (hPSCs) and immune cells remains slow, unpredictable, and often limited by silencing, low efficiency, and an inability to handle large or multiple genetic cargos. These limitations delay R&D timelines and restrict what is possible in cell therapy, disease modeling, and synthetic biology.
To overcome this, researchers developed XPRESSO, a fast, robust, and non-viral system that enables stable, high-level expression of one or multiple genes in hPSCs within days, not months.
The platform integrates a modular transposon-based vector with a powerful expression architecture containing:
• chromatin-opening elements to prevent silencing (UCOE),
• intronic and post-transcriptional enhancers for strong, uniform expression, and
• a design that supports very large and multi-component constructs (e.g., Cas9, reporters, cytokines, optogenetic channels).
XPRESSO enables multiplex engineering, maintains expression through long-term culture and differentiation, and is compatible with a wide range of cell types — including cardiomyocytes, neurons, T cells, NK cells, and more.
The result is a rapid, scalable, and cost-effective alternative to viral vectors and traditional HDR-based editing.

Advantages:

• Rapid creation of stable engineered lines (≈1–2 weeks)
• Strong, durable expression without silencing
• Supports large genes and multiplex modifications
• Non-viral and cost-efficient
• Compatible with stem cells and immune cells

Applications and Opportunities:

• Cell therapy engineering (CAR-T, CAR-NK, cytokine-modified immune cells)
• Regenerative medicine and engineered hPSC-derived tissues
• Reporter lines, disease models, and high-content drug discovery
• Cas9/Cas12-expressing platforms for functional genomics
• Robust expression of optogenetic and chemogenetic actuators for advanced functional assays

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Dr. Mor Goldfeder
Director of Business Development, life Sciences
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