Biotechnology company focused on developing gene editing therapies for serious genetic diseases.
Graphite Bio, Inc. is a clinical-stage gene editing company dedicated to advancing innovative therapies for severe and life-threatening diseases within the United States. The company focuses on pioneering treatments that harness the potential of gene editing technology to address unmet medical needs.
Graphite Bio, Inc. is at the forefront of genetic medicine with a robust pipeline that includes several promising product candidates. Among these are GPH101, a cutting-edge gene-edited autologous hematopoietic stem cell therapy designed to directly correct the mutation responsible for sickle cell disease and restore normal adult hemoglobin expression. Additionally, the company is developing GPH201 for X-linked severe combined immunodeficiency syndrome and GPH301 for Gaucher disease, a genetic disorder characterized by a deficiency in the glucocerebrosidase enzyme.
Formerly known as Integral Medicines, Inc., Graphite Bio, Inc. rebranded itself in August 2020 to better reflect its focus and ambitions in the field of genetic therapies. Since its inception in 2017, the company has rapidly evolved, leveraging cutting-edge research and development to propel its gene editing platforms forward.
Headquartered in South San Francisco, California, Graphite Bio, Inc. operates with a mission-driven approach to transforming the treatment landscape for genetic diseases. With its innovative technologies and dedicated team of researchers and scientists, the company remains committed to making significant advancements in the realm of gene therapy to benefit patients worldwide.