The landscape and trajectory of global CRISPR therapeutics

With the first regulatory approval in 2023 of a CRISPR-based therapy for sickle cell disease followed by the recent demonstration of an accelerated, personalized CRISPR treatment for congenital severe carbamoyl-phosphate synthetase 1 deficiency, CRISPR-Cas9 genome editing has progressed from a laboratory technology into a clinical reality in just over a decade. While enthusiasm for deploying CRISPR-Cas9 to treat a range of human genetic diseases continues to grow, broad clinical application remains constrained due to technical, regulatory, manufacturing and economic challenges. Here, scientists from the European Cooperation in Science and Technology (COST) action Genome Editing to Treat Human Diseases (GenE-HumDi) provide a comprehensive global overview of the CRISPR therapeutic landscape. We systematically analyze CRISPR-based therapeutic trials registered on ClinicalTrials.gov and the EU Clinical Trials Register, providing a thorough assessment of the current clinical activity and the trajectory of CRISPR technology as it advances toward routine clinical interventions.