Lexology December 18, 2023
On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved two new gene therapies for the treatment of sickle cell disease.[1] The first, Casgevy™ (exagamglogene autotemcel (exa-cel)), is the first-ever approved cell-based gene editing therapy to be used in humans, and was developed in joint partnership between Vertex Pharmaceuticals and CRISPR Therapeutics.[2] Casgevy™ is indicated for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso occlusive crises (VOCs).[3] The second, Lyfgenia™ (lovotibeglogene autotemcel (lovo-cel)), is developed by bluebird bio, and is indicated for the treatment of patients 12 years or older with sickle cell disease and a history of vaso-occlusive events (VOEs).[4]
Sickle cell disease is an inherited blood disorder caused by...
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