Coiled-coil mediated exonuclease tethering technology for the enhancement of CRISPR gene editing

ended

The discovery and implementation of CRISPR technology represents a milestone for biotechnology and life sciences in general as the genomes of virtually all organisms can be modified by this technology and have already resulted in billions of euros of added value. With this technique, selected genes can be rather easily inactivated (knock out), which is one of the key applications of CRISPR for cell-based therapy and industrial biotechnology. Any improvement of the efficiency of CRISPR therefore has very high value and potential impact. Recently we designed Cas9 DNA targeting and cleavage complex to be tethered to the ExoIII exonuclease via an engineered coiled-coil (CC) dimer, developed in the ERC AdG project MaCChines. We found that it strongly improved inactivation of target genes, even better than direct genetic fusion of Cas9-ExoIII. Since this invention has a substantial translation potential we recently filed a patent application (priority number EP19192490). The strategic frontier of CCedit is to maximise the value of this enhancement of Cas9/CRISPR technology and establish the innovation potential arising from the ERC project MaCChines in the pre-demonstration phase. Specific objectives are to: • Refine the components of the CCedit technology and its implementation for specific applications; • Establish technological viability and its placement within the framework of Cas9/CRISPR technology; • Demonstrate and select most promising implementations of this technology for applications; • Investigate the IP, FTO and policy regulations landscape and further develop the IP portfolio; • Develop the business plan and commercialisation strategy for the technology; • Disseminate information on this technology and raise the interest of companies and other potential users of this technology and obtain links to early stage funding. • Train members of the project team and local community on entrepreneurial skills in biotech