By SPC News Staff
The FDA approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients aged 12 years and older.
One of the therapies, exagamglogene autotemcel (exa-cel; Casgevy, Vertex Pharmaceuticals), is the first FDA-approved treatment to use CRISPR/Cas9, a type of genome editing technology, in humans. The second therapy, lovotibeglogene autotemcel (lovo-cel; Lyfgenia, Bluebird Bio), uses a conventional lentiviral vector for genetic