CRISPR Gene Therapy Cures Sickle Cell Disease

Following FDA approval of the first CRISPR-based gene therapy for sickle cell disease in December 2023, expanded clinical results published in 2025 showed that over 90% of treated patients remained symptom-free after two years. The therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, was declared curative in 29 of 31 patients in one study. It represented the clearest demonstration to date of CRISPR's transformative potential in genetic medicine.