In a world first, Vertex, CRISPR win UK approval for CRISPR-edited therapy to treat sickle cell disease, beta-thalassemia

Ver­tex and CRISPR Ther­a­peu­tics have won the first-ever ap­proval for a ther­a­py cre­at­ed with CRISPR gene edit­ing, as the UK drug reg­u­la­tor MHRA green­lights their treat­ment for two blood dis­or­ders.

The ther­a­py pre­vi­ous­ly known as exa-cel will now be brand­ed Cas­gevy. It is au­tho­rized to treat sick­le cell dis­ease and trans­fu­sion-de­pen­dent be­ta-tha­lassemia.

“To­day is a his­toric day in sci­ence and med­i­cine: this au­tho­riza­tion of Cas­gevy in Great Britain is the first reg­u­la­to­ry au­tho­riza­tion of a CRISPR-based ther­a­py in the world,” said Ver­tex CEO Resh­ma Ke­wal­ra­mani in a Tues­day state­ment.

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