Markets Slaughter Gene Therapy Biotech On Day of FDA Approval? Why—TrialSite Knows

Dec 20, 2023 | News

The U.S. Food and Drug Administration (FDA) approved Casgevy (exagamglogene autotemcel) on December 8, 2023. Casgevy is a gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics. It is the first medicine to be approved in the United States based on the Nobel Prize-winning technology of gene editing. Casgevy is approved to treat sickle cell disease in people aged 12 and older. The goal of the therapy is to help the body produce a fetal form of hemoglobin, which is present at birth. The adult form of hemoglobin is defective in people with sickle cell disease. Also, the FDA approved Lyfgenia is a cell-based gene therapy developed by bluebird bio. Lyfgenia uses a lentiviral vector (gene delivery vehicle) for genetic modification and is approved for the treatment of patients 12 years of age and older with sickle cell disease and a history of vaso-occlusive events.


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