Posted by Michael Wonder on 13 Feb 2024
European Commission approves first CRISPR/Cas9 gene-edited therapy, Casgevy (exagamglogene autotemcel), for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia
13 February 2024 - Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassaemia may be eligible for treatment.
Vertex Pharmaceuticals announced today that the European Commission has granted conditional marketing authorisation to Casgevy (exagamglogene autotemcel), a CRISPR/Cas9 gene-edited therapy.
