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Sickle Cell Disease

Prevalence: ~7.7M

hemoglobin

Official·OMIM:603903 Official·ORPHA232

Inherited blood disorder caused by a mutation in the beta chain of hemoglobin. Red blood cells assume a sickle shape.

Approved Therapies

Casgevy (exagamglogene autotemcel)

FDA Approved12/8/2023CRISPR-Cas9

Associated Genes

HBBHemoglobin Subunit Beta

Chromosome 11Autosomal Recessive