DNA is the very essence of almost every living thing, determining characteristics from the color of our eyes to how our metabolism works. It’s what makes us who we are. Emendo is at the forefront of cutting-edge genetic medicine, developing genome editing technology that can repair and eliminate genetic mutations in living cells that cause serious diseases or disorders.
Permanent genomic manipulation designed to edit the genome to fix a genetic disorder
Applying our expertise in protein engineering, directed evolution and optimization of designer chimeras, we are engineering gene editing solutions that are recombination-based, highly specific and have zero off-target effect.
Gene therapy v gene editing
In current terminology, a distinction is made between gene therapy and gene editing. Gene therapy provides a transient solution, introducing the correct copy of the defective gene to the cell cytosol, without affecting the genome itself. Gene editing relates specifically to permanent genomic manipulation, designed to edit the genome in order to fix a genetic disorder. Gene editing shows great promise in the biotherapeutic world, however there are still many challenges to overcome.
Emendo in gene editing
Our protein engineering experience is allowing us to engineer site-specific nuclease for an accurate, sequence-specific and efficient correction mechanism that is HDR mediated, and effective in post-mitotic cells. We are working towards a well-established platform that will enable us to address a large pool of genetic diseases, many of which have been untreatable until now.