Novel gene editing and cell therapy approaches are advancing treatment for rare diseases and oncology. Researchers developed a universal cDNA insertion strategy via gene editing for chronic granulomatous disease, restoring immune function. In glioblastoma, targeting B-cell lymphoma 6 provides a prospective therapeutic avenue. Evolveimmune's trispecific T-cell engager EVOLVE-104 received FDA IND clearance for a solid tumor phase I trial. These advances emphasize innovative biotechnology applications in precision medicine.