Advanced Gene Editing
Genespire’s Advanced Gene Editing platform enables high efficacy, permanent targeted gene integration of therapeutic genes in the genome of a patient’s blood cells, for broad applications in genetic diseases and beyond. Our targeted approach is designed to either (i) precisely replace defective genes at their natural genomic locus to restore both function and expression control, for genes where regulated expression is required or (ii) introduce therapeutic genes in a predefined chromosomal location that allows for safe and sustained long-term expression, for genes that need to be expressed at high levels and/or for which random integration is not suitable.
Our Advanced Gene Editing platform is applicable to both T-cells and Hematopoietic Stem and Progenitor Cells (HSPCs) and is extremely versatile. We optimized the technology to provide unprecedented levels of targeted integration in HSPCs thanks to our Gene Editing Enhancer Technology, for life-long expression of the therapeutic gene and sustained therapeutic effect. Our platform can be used for short and long genes, allows selection of corrected cells and is therefore suitable for a large number of therapeutic indications, in genetic diseases and beyond.