CASGEVY
Commercial- Indications
- Sickle cell disease, Transfusion-dependent beta-thalassemia
Company intelligence / Profile preview
CRISPR Therapeutics is a leading Swiss-American biotechnology company dedicated to developing transformative gene-based medicines using its proprietary CRISPR/Cas9 gene-editing platform. The company achieved a historic milestone with the global approval of CASGEVY, the first-ever CRISPR-based therapy, for the treatment of sickle cell disease and beta thalassemia in collaboration with Vertex Pharmaceuticals. Beyond its lead program, CRISPR Therapeutics is advancing a diverse pipeline of ex vivo and in vivo therapies across immuno-oncology, autoimmune diseases, cardiovascular health, and regenerative medicine. By leveraging advanced delivery technologies like lipid nanoparticles and hypoimmune cell engineering, the company aims to provide one-time, potentially curative treatments for both rare and common chronic conditions.
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