Cutting-edge cell and gene therapy developments are reshaping treatment landscapes for complex and rare conditions. The University of Nebraska pioneered the world’s first allogeneic CAR T-cell therapy administered for multiple sclerosis, showing promising neurological improvements. MD Anderson Cancer Center and Phoenix SENOLYTIX formed a global cross-licensing deal for inducible switch technologies enhancing safety and efficacy in cell and gene therapies. Meanwhile, Regenxbio reported encouraging one-year data for its Hunter syndrome gene therapy awaiting regulatory approval. These advances highlight the surge of cell and gene modalities tackling difficult diseases with transformative potential.