Stanford doctors develop first FDA-approved gene-editing treatment

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Stanford doctors develop first FDA-approved gene-editing treatment

The Food and Drug Administration (FDA) approved the gene therapy Casgevy in December, which uses CRISPR/Cas9 genome editing technology to treat sickle-cell disease (SCD) in patients twelve years and older. The drug, also called “exa-cel,” is the first gene-editing medical treatment to be FDA-approved.  Two Stanford faculty were closely involved with the process: Stanford Professor of Pediatrics Matthew Porteus M.D. Ph.D ’94 pioneered a method of…