Posted by Michael Wonder on 26 Apr 2021
Vertex and CRISPR Therapeutics announce Priority Medicines (PRIME) designation granted by the European Medicines Agency to CTX001 for transfusion-dependent beta thalassaemia
26 April 2021 - Vertex Pharmaceuticals and CRISPR Therapeutics today announced the EMA has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of transfusion-dependent beta thalassaemia.
CTX001 was previously granted PRIME designation for the treatment of sickle cell disease in 2020.
