Posted by Michael Wonder on 25 Sep 2020
CRISPR Therapeutics and Vertex Pharmaceuticals announce Priority Medicines (PRIME) designation granted by the EMA to CTX001 for the treatment of sickle cell disease
24 September 2020 - CRISPR Therapeutics and Vertex Pharmaceuticals 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 severe sickle cell disease.
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from transfusion-dependent beta thalassaemia or severe sickle cell disease, in which a patient’s haematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells.
