FDA approves two gene therapies for sickle cell disease, including first that uses CRISPR

In a land­mark mo­ment for ge­net­ic med­i­cine and sick­le cell dis­ease pa­tients, the FDA on Fri­day ap­proved not just one but two gene ther­a­pies for the dis­ease: Ver­tex Phar­ma­ceu­ti­cals’ and CRISPR Ther­a­peu­tics’ Cas­gevy, mark­ing the first-ever ap­proval of a CRISPR-based med­i­cine in the US, and Lyf­ge­nia, blue­bird bio’s lentivi­ral gene ther­a­py.

The treat­ments are ap­proved for sick­le cell dis­ease pa­tients 12 years of age and old­er who ex­pe­ri­ence painful at­tacks as­so­ci­at­ed with the dis­ease. Ver­tex will charge $2.2 mil­lion in the US for Cas­gevy, while blue­bird will charge $3.1 mil­lion for Lyf­ge­nia.

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