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Main focus: Gene therapies and gene editing for the treatment of genetic diseases
Company stage: Clinical
Diseases (gene editing): Sickle cell disease, beta-thalassemia, duchenne muscular dystrophy, lysosomal metabolic diseases
Genome editing tool: CRISPR-Cas9
Funding stage: Private
Location: Évry, Ile-de-France, France
Généthon is a French company focused on the development of gene therapies directed towards rare diseases. The company develops gene-editing based therapies as well as other types of gene therapies. Its gene-editing activities are centered around haematopoietic stem cells and blood disorders, such as sickle cell disease.