Graphite Bio, Inc., a clinical-stage gene editing company, engages in developing therapies for serious and life-threatening diseases in the United States. It offers GPH101, a gene-edited autologous hematopoietic stem cell product candidate to directly correct the mutation that causes sickle cell disease and restore normal adult hemoglobin expression; GPH201 for the treatment of X-linked severe combined immunodeficiency syndrome; and GPH301, a product candidate for the treatment of Gaucher disease, a genetic disorder that results in a deficiency in the glucocerebrosidase enzyme. The company was formerly known as Integral Medicines, Inc., and changed its name to Graphite Bio, Inc. in August 2020. Graphite Bio, Inc. was incorporated in 2017 and is headquartered in South San Francisco, California.

Graphite Bio, Inc.

Graphite Bio is a clinical-stage, next-generation gene editing company driven to discover and develop cures for a wide range of serious and life-threatening diseases. The company is pioneering a precision gene editing approach that has the potential to transform human health by achieving one of medicine’s most elusive goals: to precisely “find & replace” any gene in the genome. Graphite Bio’s UltraHDR™ gene editing platform takes CRISPR beyond cutting and harnesses the power of high-efficiency precision DNA repair, also known as homology-directed repair (HDR), to precisely correct genetic mutations, replace entire disease-causing genes with functional genes or insert new genes into predetermined, safe locations. Additionally, the company is advancing the development of novel conditioning regimens to help bring curative therapies to more patients. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, M.D., and Matthew Porteus, M.D., Ph.D.

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