Vertex Pharmaceuticals and CRISPR Therapeutics have become the first companies to secure FDA approval for a CRISPR-based gene-editing therapy.
Vertex Pharmaceuticals, in partnership with CRISPR Therapeutics, has achieved a historic milestone with the FDA approval of exagamglogene autotemcel (exa-cel, branded as CASGEVY)—a first-ever CRISPR/Cas9–based gene therapy for sickle cell disease (SCD) and, later, transfusion-dependent beta-thalassemia (TDT). This landmark regulatory green light comes roughly seven years after exa-cel’s first clinical testing and ten years after CRISPR’s Nobel-winning discovery.
From Discovery to FDA Approval
In 2015, Vertex and ...
