CRISPR Therapeutics is a clinical-stage biopharmaceutical company that develops gene-based medicines using CRISPR/Cas9 technology, which makes precise edits to a patient's DNA to permanently correct disease-causing genetic defects. The company's only approved product is CASGEVY (exa-cel), co-developed and co-commercialized with Vertex Pharmaceuticals. CASGEVY is the world's first approved CRISPR-based therapy, indicated for sickle cell disease and transfusion-dependent beta thalassemia. CASGEVY works by editing a patient's own stem cells outside the body to reactivate fetal hemoglobin production, compensating for defective adult hemoglobin. CRISPR Therapeutics earns 40% of CASGEVY's net profits, with Vertex taking 60% and leading global commercialization. CASGEVY is priced at ~$2.2M per patient in the U.S. Beyond CASGEVY, the company is building a pipeline across four areas: hemoglobinopathies, in vivo liver editing, CAR T cell therapy, and regenerative medicine. The most advanced pipeline programs include CTX310, an in vivo liver-editing therapy targeting cardiovascular lipid reduction (ANGPTL3), and zugo-cel, an allogeneic off-the-shelf CAR T therapy being studied in B-cell lymphoma and autoimmune disease. The company manufactures zugo-cel and other clinical candidates at its own GMP facility in Framingham, Massachusetts. Near-term revenue depends on CASGEVY's commercial ramp; long-term value hinges on pipeline progress, particularly in vivo programs targeting large cardiovascular populations.