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Main focus: Gene editing for the treatment of genetic diseases
Diseases: Sickle cell disease, X-linked severe combined immunodeficiency syndrome (XSCID), Gaucher disease
Gene editing tehcnology: CRISPR-Cas9
Company stage: Clinical
Funding stage: Public (NASDAQ:GRPH)
Location: South San Francisco, CA, USA
Graphite Bio is a company mainly focusing on genomic engineering of haematopoietic stem cells (HSC) using CRISPR-Cas9 technology. The company has recently initiated clinical trials for its lead programme, GPH101. The programme is being developed for the treatment of sickle cell disease, and thus, is competing with similar programmes from CRISPR Therapeutics, EdiGene and Sangamo Therapeutics. Other diseases pursued by the company include X-linked combined severe immunodeficiency and Gaucher disease.