TARGATT™ technology, a cutting-edge gene editing platform that facilitates rapid and precise integration of large DNA fragments (up to 20 kb) into a specific intergenic locus. This unique technology ensures stable integration into a transcriptionally active safe harbor site with exceptional efficiency. By utilizing an “attP” integrase recognition landing pad in conjunction with an “attB” containing donor plasmid and integrase expression, single-copy gene integration is achieved at the preselected locus.

The TARGATT™ gene editing platform is incredibly versatile, making it ideal for creating large fragment knock-in cell lines, bioproduction, and library construction. By avoiding the issues associated with random integration, such as position effect, gene silencing, and instability due to the integration of multiple transgene copies, this technology represents a significant advancement in gene editing capabilities.technology, enables fast and site-specific, stable integration of large DNA fragments (up to 20 kb) into an intergenic, transcriptionally active safe harbor locus with very high efficiency. The preselected locus is engineered to contain an “attP“ integrase recognition landing pad where single-copy gene integration occurs when used in conjunction with an “attB” containing donor plasmid and integrase expression.

The TARGATT™ gene editing platform is versatile and can be used for the development of large fragment knock-in cell lines, bioproduction, and library construction. This technology circumvents problems associated with random integration such as position effect, and gene silencing or instability due to the integration of multiple copies of the transgene.

Advantages of TARGATT™ Technology

• Site-specific knock-in at a pre-selected safe harbor locus (no random integration)
• Single copy gene insertion
• Large transgene knock-in (up to 20 kb)
• No internal genes are interrupted
• Uninformed expression
• High integration efficiency & High-level expression of the transgene
• Eliminates position effect

5 Reasons to Consider TARGATT™ Genome Engineering Technology for
Safe and Efficacious Induced Pluripotent Stem Cell Therapies

1. Specificity & Safety: unidirectional site-specific integration into a preselected locus
2. Gene Editing Efficiency: offers high efficiency for the site-specific insertion of large transgenes, achieving insertion efficiencies as high as 40% in many cell lines, including iPSCs
3. Editing In Non-Dividing Cell Lines: facilitates targeted genetic modifications in quiescent or terminally differentiated cells, unlocking novel opportunities for disease modeling, drug discovery, and tissue regeneration
4. Payload Capacity: integration of transgenes of up to 20 kb (case studies available)
5. Manufacturing & Outsourcing: avoiding the complexities associated with lentiviral vector production & FTO

It is vital to conduct a comprehensive evaluation of gene editing platforms for the development of safe and effective cell therapies. ASC’s proprietary TARGATT genome engineering platform has emerged as a highly promising solution. It offers specificity, efficiency, safety, editing capabilities in non-dividing cells, a large payload capacity, a clear IP path to commercialization, and streamlined, time-saving, and cost-effective manufacturing considerations. By leveraging the unique advantages of TARGATT, researchers can expedite the development of allogeneic iPSC-based cell therapies, paving the way for a new era of patient-centric treatment modalities.