Gene therapy allows the introduction in a gene encoding a faulty or missing protein. This approach offers potential cures for diseases that until now require life-long treatments, such as hemophilia or thalassemia
Gene Replacement Therapy
Genetic diseases are caused by defective genes. Gene replacement therapy aims to treat such diseases by introducing a corrective gene into a patient’s body; the corrective gene can be a normal copy of the defective gene, or a copy that is engineered to provide enhanced biological activity; one of our focuses for gene replacement therapy is to identify such gene variants with superior therapeutic potency and to achieve optimal and sustainable production of the functional protein once administered to the patient.
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.