Posted by Michael Wonder on 16 Nov 2023
Vertex and CRISPR Therapeutics announce authorisation of the first CRISPR/Cas9 gene edited therapy, Casgevy (exagamglogene autotemcel), by the United Kingdom MHRA for the treatment of sickle cell disease and transfusion dependent beta thalassaemia
16 November 2023 - First regulatory authorisation of a CRISPR-based gene editing therapy in the world.
Vertex Pharmaceuticals and CRISPR Therapeutics announced today that the United Kingdom Medicines and Healthcare products Regulatory Agency has granted conditional marketing authorisation for Casgevy (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene edited therapy, for the treatment of sickle cell disease and transfusion dependent beta thalassemia.
