Editas Medicine Announces Publication in Nature Biotechnology of Comprehensive SLEEK Gene Editing Technology Data
Despite major progress in achieving gene disruption with CRISPR-Cas gene editing technologies, efficient knock-in of transgenes continues to be a significant challenge for the gene editing field. To solve this challenge, SLEEK was developed to enable high knock-in efficiency with both viral and non-viral transgene formats while also ensuring robust simultaneous expression of up to four transgene cargos.
The study demonstrated that utilizing SLEEK results in the knock-in of multiple clinically relevant transgenes through a proprietary process that specifically selects only those cells containing the knock-in cargo. This process was developed by leveraging Editas Medicine’s proprietary engineered AsCas12a nuclease, which can achieve very high editing efficiency while maintaining high specificity. More than 90 percent knock-in efficiencies were observed in various clinically relevant target cells, including T cells, B cells, iPSCs, and NK cells. Additionally, SLEEK can be used to fine-tune the expression levels of transgene cargos, an important feature of next-generation cell therapies. As a demonstration of SLEEK’s potential value in clinical applications, the study authors used SLEEK to generate iPSC-derived NK cells capable of high-levels of in vivo persistence and robust tumor clearance in a solid tumor animal model.
“We are thrilled Nature Biotechnology published our paper sharing the comprehensive data on our SLEEK gene editing technology as we believe SLEEK has immense potential for gene editing drug development. As shared in the publication, SLEEK technology enables nearly 100 percent knock-in of functional transgene cargos at specific locations in the genome which may result in highly efficient multi-transgene knock-in for the next generation of cell therapy medicines,” said
About Editas Medicine
As a clinical stage genome editing company,
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Source: Editas Medicine, Inc.