VRTX - UK Approves First Crispr Gene Edited Therapy For Sickle Cell Disease Beta Thalassemia | Benzinga
The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for Casgevy (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) jointly developed the gene therapy with CRISPR Therapeutics AG (NASDAQ: CRSP).
There are an estimated 2,000 patients eligible for Casgevy in the U.K.
"Today is a historic day in science and medicine: this authorization of Casgevy in Great ...