Cellectis Publishes Nature Communications Article on a Non-Viral Gene Editing Process Enabling Efficient Gene Insertion in Hematopoietic Stem Cells

Core Insights - Cellectis has established circular single-stranded DNA (CssDNA) as an efficient non-viral DNA donor template for gene insertion in hematopoietic stem and progenitor cells (HSPCs) [1][5] Gene Editing Technology - Gene editing of HSPCs has the potential for long-term therapeutic benefits, with non-viral DNA templates emerging as promising alternatives to viral vectors like AAV6, which pose safety and efficacy concerns [2] - Cellectis has developed a robust gene insertion process using TALEN technology and CssDNA donor templates, enabling precise integration of large genetic sequences within HSPCs, thus expanding the potential of non-viral gene therapy [3] Efficiency and Application - CssDNA demonstrates 3-5 times higher knock-in efficiency than linear single-stranded DNA (LssDNA), with efficiencies surpassing 40% [9] - CssDNA can insert genes at multiple loci in HSPCs and is applicable to other therapeutic cell types, including primary T cells [9] - Comparative studies indicate that CssDNA-edited HSPCs have a higher propensity to engraft and maintain gene edits in murine models compared to AAV6-edited HSPCs [9] Company Overview - Cellectis is a clinical-stage biotechnology company focused on developing life-saving cell and gene therapies, utilizing an allogeneic approach for CAR T immunotherapies in oncology [6] - The company controls the entire cell and gene therapy value chain from start to finish, supported by in-house manufacturing capabilities [6] - Cellectis is headquartered in Paris, France, with additional locations in New York and Raleigh, NC, and is listed on both the Nasdaq Global Market and Euronext Growth [7]