Targeted therapies in neuro-oncology have shown promise in improving patient outcomes, particularly in gliomas and other brain tumors. A notable study on vorasidenib, an IDH1/2 inhibitor, demonstrated significant improvements in imaging-based progression-free survival compared to placebo, with a hazard ratio of 0.26 (ref: Mellinghoff doi.org/10.1056/NEJMoa2304194/). This highlights the potential of targeted therapies to extend the time before the need for further interventions. Additionally, the investigation of unecritinib, a multi-tyrosine kinase inhibitor, revealed a 46.9% incidence of grade 3 or higher treatment-related adverse events, emphasizing the need for careful monitoring of safety profiles in advanced non-small cell lung cancer (ref: Lu doi.org/10.1038/s41392-023-01454-z/). Furthermore, the exploration of RNA-engineered CAR T-cell therapy in autoimmune diseases like myasthenia gravis suggests a novel approach to enhance safety and efficacy in targeted treatments (ref: Granit doi.org/10.1016/S1474-4422(23)00194-1/). The development of advanced human iPSC-based models for neurodegenerative diseases, such as Parkinson's, using optogenetics to induce alpha-synuclein aggregation, represents a significant step forward in understanding disease mechanisms and testing targeted therapies (ref: Kim doi.org/10.1016/j.stem.2023.05.015/). Lastly, the role of super-enhancer-driven lncRNA LIMD1-AS1 in glioma progression underscores the importance of molecular mechanisms in targeted therapy development (ref: Chen doi.org/10.1038/s41419-023-05892-z/).