We are an innovative genetic medicines company focused on creating best-in-class gene editing therapies based on our proprietary GeneTether™ platform. Our GeneTether platform has the potential to significantly improve upon current gene editing methods by actively positioning reparative DNA fragments, which are referred to as donor DNA templates, near the location of double strand breaks in a cell’s DNA. These double strand breaks can be created by certain types of DNA-cutting tools, including CRISPR/Cas9. Donor DNA templates contain normal copies or fragments of the mutated, disease-causing genes of interest. By positioning a donor DNA template directly at the site of a double strand break, our GeneTether platform drives the DNA repair process towards homology-directed repair (HDR), a cellular repair mechanism that incorporates the genetic information of a DNA template, rather than the error-prone process of non-homologous end joining (NHEJ).
We are currently building a discovery pipeline focused on the treatment of rare, monogenic diseases of the kidney and the skin. We believe that our GeneTether platform may have broad applicability and we are exploring other potential uses, including for the treatment of genetic diseases in in organs beyond the kidney and skin. Our GeneTether platform may also allow for high fidelity engineering of cells to permanently deliver the therapeutic proteins necessary to treat certain genetic diseases.