Graphite Bio, Inc. is a clinical-stage gene editing company dedicated to developing innovative therapeutic solutions for severe and life-threatening illnesses across the United States. Its primary investigational therapy, GPH101, is an autologous, gene-edited hematopoietic stem cell product specifically engineered to directly correct the underlying mutation causing sickle cell disease, thereby restoring normal adult hemoglobin production. The company's pipeline also features GPH201, intended for the treatment of X-linked severe combined immunodeficiency syndrome, and GPH301, an experimental candidate designed to address Gaucher disease, a genetic disorder characterized by a deficiency in the glucocerebrosidase enzyme.