2023

On 8 December, FDA approves first medical application of CRISPR/Cas9 gene-editing technology in the U.S. Casgevy, (exagamglogene autotemcel), an ex vivo, exogamous (i.e., using the patient’s own blood) application of CRISPR/Cas9, was approved for treatment of sickle-cell disease in patients 12 years and older. On the same day, the FDA also approved Lyfgenia, another cell-based gene therapy (i.e., genetic modification of the patient’s blood stem cells) for patients 12 years and older with sickle cell disease (FDA News Release, 8 December 2023). The UK’s announcement preceded the FDA’s by several weeks, when the Medicines and Health Care Agency gave the green light for the use of Casgevy as a treatment for sickle cell disease and transfusion-dependent β-thalassemia. “The UK’s decision to endorse Casgevy in sickle cell disease was based on an impressive ability to eliminate severe vaso-occlusive crises — painful inflammatory attacks, often requiring hospitalization — in 28 out of 29 trial participants eligible for evaluation. It remains to be seen whether the therapy will also reduce stroke and organ damage in the long term and, crucially, extend life expectancy” (Sheridan).