Casgevy (exa-cel)
Active
Vertex Pharmaceuticals
Overview
The first approved CRISPR/Cas9 gene-editing medicine (exagamglogene autotemcel), a one-time therapy for sickle cell disease and transfusion-dependent beta thalassemia, developed and commercialized with CRISPR Therapeutics.
Approved across multiple jurisdictions and priced at ~$2.2M per one-time treatment. Vertex books the revenue (~$116M in FY2025) and is scaling a global network of authorized treatment centers; in July 2026 the US label expanded to patients ages 2+.