Inside Precision Medicine January 17, 2024
The U.S. Food and Drug Administration (FDA) has approved Vertex and CRISPR Therapeutics’ Casgevy (exa-cel), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. This is the latest sign the gene and cell therapy market is maturing. This market is already estimated to be worth $15B and expected to grow to more than $80B over the next ten years or so.
Just a few weeks ago the FDA approved Casgevy to treat patients with sickle cell disease. Simultaneously, Bluebird Bio received the FDA OK for Lyfgenia, its gene therapy for sickle cell. In November, the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) granted conditional marketing authorization for Casgevy in...
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