Innovative cell and gene therapy approaches demonstrate clinical potential. Vertex Pharmaceuticals' cell therapy zimislecel shows insulin independence in type 1 diabetes patients entering pivotal trials. Adeno-associated virus RNAi targeting ataxin-2 extends survival in TDP-43 mouse models relevant to ALS and FTD. Additionally, CAR T-cell therapies are being explored for neurological disorders, while organ-chip models using patient iPSCs reveal early pathology in ALS. These developments underscore progress toward personalized and regenerative medicine paradigms in complex diseases.