Europeans Medicines Agency (EMA) grants PRIME designation to CTX001 for transfusion-dependent beta thalassemia

PRIME designation for this investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy was granted based on clinical data from ongoing Phase 1/2 trials (CLIMB-111,121 and 131) of CTX001 in patients with transfusion-dependent beta thalassemia.

Biospace Inc.