Vertex Pharmaceuticals and CRISPR Therapeutics have scored another regulatory approval for Casgevy (exagamglogene autotemcel) after the European Commission granted conditional marketing ...

However, Casgevy and other gene therapies for sickle cell disease are complex, requiring nine months to a year to complete, including chemotherapy and weeks of hospitalization.

Vertex already has an approved treatment for sickle cell disease and transfusion-dependent beta thalassemia called Casgevy. However, Casgevy is an ex vivo treatment, meaning patients’ cells are ...

“We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with Casgevy ... the overall data package we've been able to generate ...

In December, Vertex's CASGEVY gained regulatory approval in the UAE for sickle cell disease and transfusion-dependent beta-thalassemia. Suzetrigine for moderate-to-severe acute pain awaits an FDA ...