Key Points CRISPR Therapeutics made worldwide headlines coming into 2024, receiving the first-ever FDA approval for a gene editing therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Next-gen gene editing will evolve Cas9 from molecular scissors to a molecular UPS truck delivering cargo to a specific part of the genome. CTX-211 is in Phase 1 clinical trials for treating Type 1 diabetes (T1D) with the goal of enabling patients to produce their insulin instead of relying on ...