Genome Editing Therapy

Genome editing technologies enables permanent modifications to the genome by harnessing the activities of certain enzymes (e.g. nucleases and integrase) and invoking DNA repair mechanisms at specific locations in the genome. As a therapeutic modality, genome editing can correct a defective gene, disrupt a deleterious gene, or target a therapeutic gene to a precise location in the genome for safe and optimal expression of the therapeutic protein. We are applying the optimized CRISPR-Cas9 and in-house-developed TARGATT™ gene editing technologies for therapeutic development, focusing on improving both the potency and specificity of editing.