EMA PRIME status granted for Vertex, CRISPR Therapeutics’ gene therapy CTX001
Vertex Pharmaceuticals and CRISPR Therapeutics’ gene therapy CTX001 has been granted a Priority Medicines (PRIME) designation from the European Medicines Agency (EMA).
CTX001 is an investigational CRISPR/Cas9 gene-edited therapy for the treatment of transfusion-dependent beta thalassemia (TDT). Previously, the gene therapy was also granted a PRIME designation for the treatment of sickle cell disease (SCD).
A PRIME designation provides early support to the developers of ‘promising medicines’ in a bid to optimise development plans and accelerate evaluations to improve patient access.
The most recent PRIME designation for CTX001 is based on clinical data from an ongoing Phase I/II trial of the gene therapy in patients with TDT.
Patients enrolled in this trial have their hematopoietic stem and progenitor cells collected from peripheral blood.
Read more: http://www.pharmatimes.com/news/ema_prime_status_granted_for_vertex,_crispr_therapeutics_gene_therapy_ctx001_1368443
CTX001 is an investigational CRISPR/Cas9 gene-edited therapy for the treatment of transfusion-dependent beta thalassemia (TDT). Previously, the gene therapy was also granted a PRIME designation for the treatment of sickle cell disease (SCD).
A PRIME designation provides early support to the developers of ‘promising medicines’ in a bid to optimise development plans and accelerate evaluations to improve patient access.
The most recent PRIME designation for CTX001 is based on clinical data from an ongoing Phase I/II trial of the gene therapy in patients with TDT.
Patients enrolled in this trial have their hematopoietic stem and progenitor cells collected from peripheral blood.
Read more: http://www.pharmatimes.com/news/ema_prime_status_granted_for_vertex,_crispr_therapeutics_gene_therapy_ctx001_1368443
