Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY (Exagamglogene Autotemcel), by the UK MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
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Vertex Pharmaceuticals and CRISPR Therapeutics have received conditional marketing authorization from the UK MHRA for CASGEVY, a CRISPR/Cas9 gene-edited therapy for sickle cell disease and transfusion-dependent beta thalassemia. The treatment is approved for patients 12 years and older who lack a matched stem cell donor, with an estimated 2,000 eligible patients in the UK.
November 16, 2023 | 7:29 am
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CRISPR Therapeutics' collaboration with Vertex Pharmaceuticals yields UK MHRA authorization for CASGEVY, a gene-edited therapy for blood disorders.
The approval of CASGEVY is a significant achievement for CRISPR Therapeutics and its partnership with Vertex, likely to have a positive effect on its stock due to the potential for new revenue streams.
CONFIDENCE 90
IMPORTANCE 75
RELEVANCE 80
POSITIVE IMPACT
Vertex Pharmaceuticals received UK MHRA authorization for CASGEVY, a gene-edited therapy for SCD and TDT, potentially opening a new market segment.
The authorization of CASGEVY in the UK could lead to increased market penetration and revenue for Vertex Pharmaceuticals, positively impacting its stock price in the short term.
CONFIDENCE 90
IMPORTANCE 75
RELEVANCE 80