Case Study: Advancing an HIV Clinical Program for CCR5 Gene Disruption

Precision genome engineering requires technologies that allow efficient and reproducible delivery of DNA, mRNA and RNP-based reagents into a range of primary cells and stem cells. In addition, clinical gene editing requires a transfection platform that is GMP-compliant and scalable to accom- modate billions of cells in a single transfection.

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Case Study: CRISPR mRNA & RNP Delivery

Demonstrating high efficiency delivery with low primary cell toxicity at clinical-scale — all critical parameters for commercial production of quality, clinically-active genetically engineered cells.

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