Applied StemCell, Inc. (ASC), a leading gene editing company focused on the development of enabling tools for cell and gene therapy has been awarded close to $2.0 million US dollars in a Small Business Innovation Research (SBIR) grant from the NIH. This fast track Phase I/II grant award will support Applied StemCell (ASC) to advance and improve the efficiency of its proprietary TARGATT™ integrase technology for human gene therapies (referred to as the hTARGATT™) in a three part objective: (1) Develop and improve the efficiency of the TARGATT™ technology in human cell lines including primary cells and stem cells; (2) Develop therapeutic transgenes and demonstrate optimal integration efficiencies as a functional demonstration of the platform’s utility for ex vivo therapeutic applications; (3) Differentiate the above mentioned stem cells to desired somatic lineages and demonstrate regulatory induction of the transgenes.
Applied StemCell (ASC)’s approach will take advantage of the site-specificity and large DNA insertion capability of the serine integrase, PhiC31 (φC31) and engineer it to insert exogenous DNA into a defined and safe harbour locus in the human genome. The scope of the grant will also cover an unbiased detection and characterization of potential off-target insertion sites to ensure safety of this platform for therapeutic gene editing.
This study will provide useful insight into the applicability of the hTARGATT™ Gene Therapy platform and build the foundation for its use in gene therapy for potentially ten thousand monogenic human diseases.
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Marketing Director, Applied StemCell, Inc.