Zug-based biotech company CRISPR partnered up with Vertex to provide CRISPR/Cas9 gene-edited therapy for patients with inherited blood disorders, affecting more than 100 million people worldwide. They now received a conditional regulatory marketing authorization for CASGEVY, the first CRISPR-based gene-editing therapy in the world.
CRISPR Therapeutics, a gene editing company focused on developing transformative gene-based medicines using its CRISPR/Cas9 platform, and Vertex, a global biotechnology company that invests in scientific innovation to create transformative medicines against serious diseases, developed CASGEVY, a CRISPR/Cas9 based gene-editing therapy for the treatment of inherited blood disorders, namely sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in eligible patients aged 12 and older.
Sickle cell disease (SCD) is an inherited blood disorder characterized by misshapen “sickled” blood cells, which can cause painful blood vessel blockage and can result in acute chest syndrome, stroke or symptoms of heart failure. Current treatments mostly include monthly blood transfusions, significantly impacting patients’ quality of life. Transfusion-dependent beta thalassemia (TDT) is an inherited blood disease causing anemia, fatigue and shortness of breath. Patients require regular blood transfusions, which can lead to unhealthy iron buildup. Traditionally both diseases necessitate lifelong treatment, ultimately resulting in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity.
CASGEVY is a genetically modified cell population that contains human hematopoietic stem and progenitor cells edited ex vivo by CRISPR/Cas9. The U.K. Medicines and Healthcare products Regulatory Agency’s (MHRA) authorizes this treatment for eligible patients with SCD aged 12 and older, who experience recurrent vaso-occlusive crises (VOCs) and have the βS/βS, βS/β+ or βS/β0 genotype, and for patients with TDT. This comes with the condition that for both types of patients, hematopoietic stem cell transplantation is appropriate, and a human leukocyte antigen matched related hematopoietic stem cell donor is not available. The estimated eligible patient population doe CASGEVY in the U.K. alone is around 2’000 individuals.
Conditional authorizations by the MHRA are granted for medicines addressing serious and life-threatening diseases where comprehensive data is not yet complete but is expected to become available soon. These authorizations are valid for one year and are subject to annual renewal with ongoing regulatory review.
For CRISPR Therapeutics and Vertex this authorization is a scientific milestone, underscoring the transformative potential of the CRISPR/Cas9 technology, positioning both companies at the forefront of personalized healthcare solutions. This success also paves the way for future collaborations in the field of gene editing. CASGEVY is now also under review by the European Medicines Agency, the Saudi Food and Drug Authority, and the U.S. Food and Drug Administration (FDA), giving hope to thousands of eligible patients.
(SR/ press release)
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