Gene editing with ARCUS has the potential to deliver one-and-done treatments for genetic diseases.
In vivo gene editing has the potential to permanently cure genetic diseases. Unlike some editing technologies that have been designed as research tools and subsequently engineered for therapeutic use, ARCUS is a precise and versatile genome editing technology with the distinct potential to insert, delete, or repair disease-causing DNA in a broad spectrum of genetic diseases.
Gene editing enzymes are large molecules that cannot disperse across the body like traditional small molecule drugs. This means that therapeutic genome editing is not a whole-body event, but one that targets specific cells and tissues where the gene that requires correction is active. It also means the therapeutic potential of any gene editing tool is tied to its ability to reach target cells and tissues and complete complex edits safely and efficiently.
Learn more about our preclinical in vivo gene editing pipeline ›