iTOP Technology for Gene Editing

NTrans Technologies BV was founded in 2015 based on a proprietary platform technology for the intracellular delivery of bioactive molecules developed at the Hubrecht Institute of the Royal Netherlands Academy of Sciences. The iTOP intracellular delivery technology is based on a combination of small molecule compounds which forces the uptake of therapeutic proteins into the target cell where they can exert their action (D’Astolfo et al. 2015, Cell 161: 674-690). Important aspects of the iTOP delivery method include the unprecedented efficiency (>95% of the target cells are transduced), the ability to target cell types that are notoriously difficult to manipulate, such as primary (stem) cells. Furthermore, iTOP is a viral free cell targeting method that works both in vitro and in vivo. iTOP delivery of CRISPR-based gene editing systems allows efficient gene editing in therapeutically relevant cells, both ex vivo and in vivo. The iTOP delivery method is an essential technology in unlocking the therapeutic application of CRISPR/Cas gene editing tools. It finds application in personalized gene-editing therapies for the treatment of cancer and the treatment of genetic diseases.