First Regulatory Authorization For A CRISPR-based Gene-editing Therapy

(RTTNews) - Vertex Pharmaceuticals Incorporated (VRTX) and CRISPR Therapeutics (CRSP) Thursday said UK's Medicines and Healthcare products Regulatory Agency (MHRA) authorized their gene-editing therapy Casgevy for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in patients aged 12 years and older.

This makes the first regulatory authorization of a CRISPR-based gene-editing therapy in the world.

Sickle cell disease is a group of inherited red blood cell disorders. Beta thalassemia is also an inherited blood disorder, in which the body doesn't make enough hemoglobin.

Two clinical trials of Casgevy in SCD and TDT had met their respective primary goals of becoming free from painful blood vessel blockages or transfusion independent for at least 12 consecutive months.